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Posts by category
- Category: Uncategorized
- NORD’s Rare Caregiver Respite Program Featured in BioNews Service Newsletters
- Head of the Herd: Lanie Etkind, Executive Director, Familial Dysautonomia Foundation
- Rare Action Network’s Maria Bellefeuille Interviewed by Patient Empowerment Network Leading up to Living Rare Forum
- #NORDinthenews: NORD’S Mary Dunkle interviewed on WFAN Sports Radio on all things rare disease
- Students for Rare Featured Student: Viridiana Murillo
- #NORDinthenews: NORD Summit content featured in two new articles
- NORD releases statement on Administration’s recent changes to medical deferred action policy
- Voices of Rare Cancer: Mackenzie’s Story
- Read the Inspiring Personal Story of a NORD Member Organization Leader
- NORD Launches New Program Aimed at Providing Caregivers with Relief
- NORD Publishes Report on Post-Transplant Lymphoproliferative Disease
- Moving documentary on Joshua Frase Foundation, Nibs – the matriarch of the first ever MTM dog colony, and the power of patient advocacy
- Changing the Landscape of Rare Disease Research
- NORD and Neurology Reviews Publish Special Report
- President Obama Signs Advancing Hope Act
- NORD in the News: Financial Times Highlights NORD’s Training and Advice for Family Foundations
- Dr. Arthur Caplan: 2016 Rare Impact Award Honoree
- Dawn Laney: 2016 Rare Impact Award Honoree
- Debra Miller: 2016 Rare Impact Awards Honoree
- Happy Mother’s Day from NORD!
- NORD Announces New VP of Patient Services and General Counsel
- Member Spotlight: Cure SMA
- Requests for Proposals
- NORD Issues Statement on FDA’s New Associate Director for Rare Diseases
- Member Spotlight: WSCTF
- Member Spotlight: AGMD
- Member Spotlight: CdLS Foundation
- Member Spotlight: AMEN Support
- People Magazine Turns Spotlight on Rare Diseases with Personal Stories from Country Music Star Randy Rogers and Model Lauren Wasser
- Rare Disease is a Common Cause
- 2015 Portraits of Courage
- 2015 Portraits of Courage Honoree, Lori Sames
- 2015 Portraits of Courage Honoree, Yusuf Patel
- NORD Volunteer Sheds Light on CdLS for CdLS Awareness Day
- 2015 Portraits of Courage Honorees, Glenn and Cara O’Neill
- 2015 Portraits of Courage Honoree, Savannah Hollis
- 2015 Portraits of Courage Honoree, Sophia Hanson
- 2015 Portraits of Courage Honoree, Bailey Gribben
- 2015 Portraits of Courage Honoree, Tony Ferrandino
- 2015 Portraits of Courage Honoree, Laura Crandall
- 2015 Portraits of Courage Honoree: Emily Argersinger
- Get to Know Portraits of Courage Honoree, Devin Alvarez
- Running for Rare Diseases Takes On Boston Marathon
- Announcing the 2015 Portraits of Courage Honorees
- Can Crowdfunding Help Cure a Rare Disease? Cure Black Bone Disease: Time Is Running Out.
- Rare Disease Day 2015 was the biggest and most impactful yet!
- FDA Commissioner on Medical Product Innovation
- NORD’s Paul Melmeyer Authors Piece in NCC Collaborator about State-Based Advocacy
- Today is Rare Disease Day 2015
- NORD Announces #1in10 Campaign to Raise Awareness for Rare Disease Day® on February 28
- Join NORD to Celebrate Rare Disease Day
- Advancing Research: NORD Partners with Lundbeck for 6th Raise Your Hand Campaign
- Partnering with Hemophilia Federation of America for Rare Disease Day and Hemophilia Awareness Month
- Featured NORD Member Org: CTF
- Category: Press Releases
- NORD Announces This Year’s Heroes of Rare Disease: The 2021 Rare Impact Award Honorees
- How Will You Show Your Stripes in 30 Days? Join NORD in Spreading Awareness for Rare Disease Day®, February 28
- NORD State Report Card Grades States on Policy Issues Critical to Rare Disease Patients
- NORD and Partners Host FDA, CDC Leaders in Webinar Addressing Rare Disease Community on COVID-19 Vaccines
- NORD Releases Report on the State of Rare Disease Diagnosis, Care and Treatment in America Over Three Decades
- NORD Launches Project RDAC, Announces Inaugural Meeting on December 16
- NORD Drives Rare Disease Progress Through Patient-Powered Data With IAMRARE™ Registry Members
- NORD Announces RareLaunch Workshops to Support Those Seeking to Start Rare Disease Nonprofits or Research Programs
- RDCA-DAP First Year Milestones Highlighted at Virtual Workshop
- NORD Highlights New Era of Innovation and Public Health Awareness in Virtual 2020 Rare Diseases and Orphan Products Breakthrough Summit
- NORD Launches Innovative Natural History Study on Metachromatic Leukodystrophy (MLD)
- A Cure In Sight, in partnership with the UCSF Ocular Oncology Center and NORD, Launches Landmark Ocular Melanoma Natural History Study
- NORD Joins Coalition Representing Millions of Patients to Release Telehealth Principles
- Rare Cancer Day 2020 To Take Place on September 30
- 2020 NORD Summit to Feature Leading Experts in Public Policy, Patient Advocacy, Rare Disease Research and Regulatory Science
- NORD Awards COVID-19 Rapid Response Seed Grants to Rare Disease Patient Organizations Impacted by the Pandemic
- CZI Grants to NORD and EURORDIS Support Organizational Capacity Building and Rare Disease Day Campaigns
- NORD’s Jayne Holtzer Rare Disease Research Grants Program Continues Its Commitment to Rare Disease Research with New Funding Opportunities
- NORD Supports Rare Disease Nonprofits Impacted by COVID-19 with New Rapid Response Leadership Series
- NORD Awards Eleven New Grants in Eight Disease States for Rare Disease Research
- In Support of Rare Disease Patients Impacted by COVID-19, NORD Launches Premium and Limited Medical Relief Program
- New Community Survey Findings from NORD Reveal Significant Impact of COVID-19 on Americans Living with Rare Diseases
- NORD Launches Financial Assistance Program for Rare Disease Community Members Impacted by COVID-19
- NORD Launches Natural History Study for Undiagnosed Rare Diseases
- NORD Introduces This Year’s Rare Disease-Fighting Super Group: The 2020 Rare Impact Award Honorees!
- NORD State of the States Report Shows Key Policy Improvements Were Achieved by States in 2019, But More Progress Is Needed to Support Americans Living with Rare Diseases
- NORD Joins with Pyruvate Kinase Deficiency Community to Publish Historic “Voice of the Patient” Report
- NORD Issues a Rare Decree: Show Your Stripes™ for Rare Disease Day®!
- NORD Welcomes Two New Members to Board of Directors
- Innovative Data and Analytics Platform to Accelerate Drug Development for Rare Diseases
- Mark Your Calendars – October 1 is Rare Cancer Day!
- FUNDED BY FDA, C-PATH and NORD TO LAUNCH RARE DISEASE DATA ANALYTICS PLATFORM
- 2019 NORD Rare Summit to Feature Caregivers Who Have Moved from Inspiration to Action
- NORD Launches Digital Education Initiative with PlatformQ Health
- NORD AND TRIO HEALTH PUBLISH NEW BOOK TO PROVIDE CRITICAL PATIENT INSIGHTS INTO THE BURDEN OF LIVING WITH AND TREATING RARE DISEASES
- National Gathering of the Rare Disease Community and Health Professionals Launched with 2019 Living Rare, Living Stronger | NORD Patient & Family Forum Featuring the Rare Impact Awards
- NORD Releases Principles for Assessing Proposals Designed to Lower the Cost of Prescription Drugs in the United States
- NORD Research Grant Program Celebrates 30th Anniversary, Funding Opportunities Announced
- NORD IAMRARE DHPS Patient Registry Launched
- The 2019 Rare Neurological Disease Special Report Is Now Available
- NORD Announces Honorees for 2019 Rare Impact Awards
- NORD IAMRARE GBS|CIDP Patient Registry Launched
- NORD IAMRARE APS Type 1 Patient Registry Launched
- NORD Issues New Rare Disease DayⓇ Rallying Cry: Show Your Stripes™!
- NORD Expands and Enhances its Rare Disease Database®, a Primary Resource for Millions of People Affected by Rare Diseases
- Updated Study Analyzes Use and Cost of Orphan Drugs
- Patient Advocates Warn Against New Insurance & PBM Policy That Increases Patient Out-of-Pocket Drug Costs
- NORD IAMRARE HI Global Registry Launched
- Five Years of Patient Registry Success: NORD’s IAMRARE™ Registry Program Celebrates New Partnerships and Models of Engagement
- NORD Receives Grant from Anthem Foundation for Expansion of Rare Disease Database®
- NORD Announces Ten New Rare Disease Registries, Thanks to Multi-Year Grant from Shire
- 2018 NORD Rare Summit to Feature Distinguished Speakers Discussing a New Era of Patient-Focused Innovation
- Healthline and NORD Announce Recipients of 2018 Stronger Scholarships
- NORD’s RareEDU™ Launches Video Addressing a Topic Vital to Today’s Rare Disease Community, Gene Therapy: Your Questions Answered
- NORD Launches RareInsights, New Initiative to Support Data-Driven Advocacy, and Presents 5 Myths About Orphan Drugs and the Orphan Drug Act
- New Research Funding Opportunities Available from NORD
- Neurology Reviews® and NORD Publish the 2018 Rare Neurological Disease Special Report
- NORD Rare Action Network® 2018 State Report Card
- NORD to Collaborate with FDA on Pilot Patient Engagement Activity
- NORD Launches 7,000 Mile Rare Movement
- NORD and the Orphan Drug Act Celebrate 35th Anniversaries
- Rare Disease Advocates Host a Save the Orphan Drug Tax Credit Rally
- More Than 200 Patient Groups Call On Congress to Reinstate the Orphan Drug Tax Credit in the Tax Cuts and Jobs Act
- NORD Releases New Documentary called “Good Morning Peyton”
- New Study Examines Use and Cost of Orphan Drugs
- Registration Open!
- NORD Announces Three New Hires to Lead Research, Education and Business Development Programs
- NORD Issues Statement Opposing the “Graham-Cassidy” ACA Replacement Plan
- NORD Publishes New Rare Disease Report on Shprintzen Goldberg Syndrome (SGS)
- NORD Publishes New Report on Sitosterolemia
- NORD Issues Statement on the Senate passage of the Food and Drug Administration Reauthorization Act of 2017
- NORD Issues Statement on the Better Care Reconciliation Act of 2017
- NORD Publishes New Rare Disease Report on CARD9 Deficiency
- NORD Releases Statement on Orphan Drug Assessment & Pricing Summit
- Patient Advocacy Organizations Outline Joint Position on Expanded Access to Experimental Drugs for Seriously Ill Patients
- Leading Patient Advocacy Groups Stand Together to Oppose American Health Care Act
- NORD Issues Statement Opposing ‘MacArthur Amendment’, the Latest Version of the American Health Care Act
- NORD Names New Director of Membership
- International Pemphigus & Pemphigoid Foundation Launches Largest-Ever Study of Pemphigus and Pemphigoid
- NORD and Friends of Cancer Research Issue Joint Statement In Advance of this Week’s Congressional Hearings on FDA User Fees
- NORD Joins 86 Organizations to Call on Congress to Protect Medicaid
- NORD Issues Statement Opposing the Preserving Employee Wellness Programs Act (H.R.1313)
- NORD Announces Honorees for the 2017 Rare Impact Awards
- NORD Issues Statement as House Committees Review the American Health Care Act
- NORD Issues Statement on Release of the American Health Care Act
- Eleven Major Patient Groups Outline Joint Position on Health Care Reform
- Musical Act Announced for Rare Impact Awards on May 18th
- NORD Issues Statement on President Trump’s Address to Congress
- NORD Unveils “Do Your Share for Rare,” Year-long Campaign for Rare Disease Public Awareness
- NORD Awards New Research Grants for Rare Disease Research
- NORD Publishes Report on Spontaneous Intracranial Hypotension
- Orphan Drugs Represent 41 Percent of All New Medications
- First Natural History Registry for Necrotizing Enterocolitis
- Largest-Ever Study of SYNGAP1 (MRD5), Linked to Autism, was Launched
- President Signs 21st Century Cures Medical Innovation Bill Into Law
- State Report Card to Help People with Rare Diseases
- Senate Passes Landmark 21st Century Cures Act
- NORD Ready to Address New Challenges Based on Election Results
- Fibrous Dysplasia Foundation and NORD Launch Cutting-Edge Study of Fibrous Dysplasia/McCune-Albright Syndrome
- NORD Publishes Physician Guide to Cutaneous T-Cell Lymphoma
- Nominations Open for NORD’s 2017 Rare Impact Awards
- Rare Diseases & Orphan Products Breakthrough Summit Speakers
- A New Partnership that Helps Raise Awareness of Rare Diseases
- Senate Passes Short-Term Agreement to Accelerate Treatments for 15 Million Sick Children with Rare Diseases
- NORD Converges on Capitol Hill for Rally for Medical Research Hill Day to Advocate for Increased Funding for the National Institutes of Health (NIH)
- FDA Commissioner Dr. Robert Califf to Speak at NORD Summit in October
- NORD Publishes Physician Guide to Mitochondrial Myopathies
- NORD Applauds Senate for Authorizing Medical Foods Coverage for the Military in National Defense Authorization Act (S.2943)
- NORD Appoints Genetics and Metabolic Specialist Marshall L. Summar M.D. as Chairman of the Board
- NORD to Hold Rare Diseases Roundtable
- NORD Website Reaches 1 Million Monthly Visits
- NORD and Trio Health to Provide Real-World Insights to Improve Access and Quality of Care for More Than 7,000 Rare Diseases
- NORD Announces 20 Rare Disease Patient Groups Selected to Develop Natural History Studies as Part of FDA Cooperative Agreement
- NORD Announces 2016 Rare Impact Award Recipients to be Honored on May 17
- Running for Rare Named an Official Charity Partner of the 2016 TCS New York City Marathon Set for Sunday, November 6, 2016
- Running for Rare Marathon Team Expands Under NORD
- NORD Issues Statement on New FDA Commissioner Robert Califf, M.D.
- FDA Approves 21 Orphan Drugs for Rare Diseases in 2015, Sets Record for Second Consecutive Year
- Get Involved in Your State on Rare Disease Day
- NORD Developing 20 Natural History Studies for 20 Rare Diseases
- Rare Disease Day 2016 Theme and Slogan Announced!
- NORD Issues Statement on NIH Appointment of New Director for Office of Rare Diseases Research
- New Video from NORD Promotes Awareness of Rare Movement Disorder Neurogenic Orthostatic Hypotension
- Impact of the Orphan Drug Tax Credit on Treatments for Rare Diseases
- Global Voice for Rare Disease Patients Launches Today: Rare Diseases International
- NORD Issues Statement on Today’s Approval of the 21st Century Cures Initiative
- The Will to Live and the Strength for a Cure
- Category: Featured News
- National Attention for Rare Disease Day Airs on Lifetime Television
- Rare Disease Patient Advocate Tristan Lee Shares His Story with WebMD for Rare Disease Day
- NORD Offers Emergency Relief to Rare Disease Patients and Families Impacted by Natural Disasters
- Head of the Herd: Chandra Clark, VHL Alliance
- NORD Supports California Attorney General Xavier Becerra’s Nomination as Next Secretary of Department of Health and Human Services (HHS)
- NORD and Other Patient Advocacy Groups Issue Letter on Lack of Clear COVID-19 Vaccination Prioritization Guidance to the States for Rare Disease Patients
- NORD Featured in BioNews Service Article on Rare Disease Day Happenings
- NORD Launches Rare Disease Educational Support Program
- NORD Featured in February 2021 Issue of PharmaVOICE
- Statement from NORD on Dr. Janet Woodcock, Acting Commissioner of the US Food and Drug Administration (FDA)
- NORD Partners with Frontline Medical Communications on Publications for Medical Professionals in Oncology and Rheumatology
- The End of Surprise Medical Billing: NORD Applauds Bipartisan Progress to Protect Millions of Rare Disease Patients from Unexpected and Financially Crippling Medical Expenses
- STAT Features NORD in Article on the Continued Threat PPE Shortages Pose to Some Rare Disease Patients
- NORD Students for Rare Member Provides Her Perspective on “Unlocking the Power of Health Care Data” Summit Panel
- In Final Installment of Video Series, Patient Advocacy Group Leaders Share Thoughts on the Importance of Participation in RDCA-DAP
- Patient Advocacy Group Leaders Tell How Data Sharing Can Help with Understanding Rare Diseases in New Video
- New Video Series Featuring Patient Advocacy Group Leaders, “The Importance of Patient-Reported Data to Progress in Rare Disease,” Launched by NORD, C-Path
- NORD and Partners Publish Abstract Based on Web-Based Hemophilia A Educational Initiative for Patients, Caregivers and HCPs
- Connecticut Magazine Features NORD in Article on CT Nonprofits
- Telehealth Report from NORD Covered in Managed Healthcare Executive
- Head of the Herd: Charlene Son Rigby, President and Cofounder, STXBP1 Foundation
- NORD & C-Path Present 3-Part Video Series on Challenges of Rare Disease Drug Development, How RDCA-DAP Can Help
- NORD and Coalition Representing Millions of Patients Urge Policymakers to Adopt “Patient-First” Policy Agenda
- NORD and Patient Advocacy Groups Host an EL-PFDD Meeting on Krabbe Disease
- NORD Issues Report on Ensuring Access to Telehealth for Rare Diseases
- NORD mentioned, RareInsights fact sheet featured in Psychology Today article
- NORD and C-Path Release New Video, “RDCA-DAP: Shortening the Timeline for Developing New Treatments for Rare Diseases”
- Quality of Life and Butterfly Wishes
- Head of the Herd: Jennifer Canvasser, Founder and Director, Necrotizing Enterocolitis (NEC) Society
- NORD and FDA Co-Host Public Listening Session, Focusing on COVID-19 and Rare Diseases
- Nebraska Medicaid Expansion Is Progress for Rare Disease Patients, Highlights Critical Need to Support Medicaid During COVID-19
- Voices of Rare Cancer: Courtney’s Story
- TODAY IS RARE CANCER DAY! Voices of Rare Cancer: Lisa’s Story
- Newborn Screening: The HCP Perspective on the Importance of Confirmatory Testing and Collaboration
- Voices of Rare Cancer: Jan’s Story
- Head of the Herd: Susan Thornton, CEO, Cutaneous Lymphoma Foundation
- NORD and C-Path Release Video on Rare Disease Challenges in Each Step of the Drug Development Process
- US Senator Richard Blumenthal (D-CT) releases statement of support regarding Rare Cancer Day
- NORD and KrabbeConnect Launch KrabbeCURES to Study Globoid Cell Leukodystrophy
- Voices of Rare Cancer: Emily’s Story
- Head of the Herd: Amber Freed, Founder and CEO, SLC6A1 Connect
- A Newborn Screening Reflection: Sarah’s Story
- Voices of Rare Cancer: Jeremy’s Story
- #RareCancerDay is September 30! NORD will share facts, information and patient stories throughout the month.
- September is Newborn Screening Awareness Month
- Head of the Herd: Deb Ayres, President, The Avalon Foundation
- New Video Series on the 5 Steps of the Drug Development Process and How RDCA-DAP Can Help
- NORD CEO Affirms Science Should Guide FDA’s Work
- NORD Releases Four Guiding Principles on Telehealth Issues
- Head of the Herd: Katie Wright, Director, The VEDS Movement (a division of The Marfan Foundation)
- NORD Included in Parade.com Article on Rare Autoimmune Diseases
- Head of the Herd: Seth Rotberg, Co-founder, Our Odyssey
- Head of the Herd: Taylor Kane, Founder and President, Remember the Girls
- The NORD Policy Team Wants to Hear About Your COVID-19 Telehealth Experience
- NORD Issues Statement on Recently Introduced “Promising Pathway Act”
- FDA Oncology Center of Excellence Listening Session with NORD: A Patient Perspective
- The Motley Fool Chooses NORD as One of Three Nonprofits Worthy of Donations
- NORD’s New Genome Editing Videos Address Patient/Caregiver Questions
- Head of the Herd: Beverley Francis-Gibson, President and Chief Executive Officer, Sickle Cell Disease Association of America
- Rare Reflections: Jacob’s Story
- 2020 Rare Diseases & Orphan Products Breakthrough Summit Will Be a Virtual Event, Oct. 8-9
- NORD Supports the Black Community and An Equal, Inclusive, Safe, Healthy Future for All Americans
- New video from NORD and C-Path addresses the question: “What is a Registry?”
- Head of the Herd: Emile Najm, KAT6A Foundation
- NORD to Host an Externally-led Patient-Focused Drug Development Virtual Meeting on Krabbe Disease, Thursday, October 29
- Infographic Illustrates Effects of the COVID-19 Pandemic on Rare Disease Patients and Families
- Historic “Voice of the Patient” Report on Pyruvate Kinase Deficiency Now Available on FDA Site
- NORD Joins 400+ Groups to Prevent Discrimination During COVID-19 Triaging
- The 2020 Living Rare, Living Stronger Forum Will Now Be Presented As A Virtual Event
- NORD’s Director of Research Programs Publishes Article on Rare Disease Research in the 2020 Rare Neurological Disease Special Report
- Update on NORD’s Recent Advocacy Work on COVID-19 for the Rare Community
- NORD and FMC’s Gene Therapy Survey: A Call to Action for Healthcare Professional Education
- Findings from NORD and Frontline Medical Communications Survey Highlight Gene Therapy Knowledge Gaps Among HCPs
- Fanconi Anemia Research Fund and NORD Launch Natural History Study of Fanconi Anemia
- Neurology Reviews and NORD Publish Annual Rare Neurological Disease Special Report
- NORD Releases 2019 Annual Report, “At the Heart of Progress in Rare Disease”
- NORD and EURORDIS-Rare Diseases Europe Issue Joint Statement on COVID-19 and Orphan Drug Legislation
- Pharmaceutical Medicine Publishes Paper by NORD Research Team as a Leading Article
- NORD Welcomes Prashant Goel as Vice President of Information Technology
- NORD Member VHL Alliance Held Jeffersonian Dinner in Early March
- US News & World Report Features NORD and Dr. Marshall Summar in “What To Do If Your Child Is Diagnosed with a Rare Disease”
- COVID-19 Resources for Non-profit Leaders and the Community
- Head of the Herd: Michele Sloan, Foundation to Fight H-abc
- NORD Announces Important Update Regarding the 2020 Living Rare Forum
- #NORDinthenews – Article on Facts Around Rare Disease Features NORD’s Kristen Angell and Vanessa Boulanger
- Rare Disease Day 2020 Advocacy Events Recap
- Head of the Herd: Akiva Zablocki, The Hyper IgM Foundation
- NORD’s Peter L. Saltonstall on Coronavirus Prevention and Risk for the Rare Community
- Savannah’s Story in honor of Rare Disease Day
- Ashlee’s Story in honor of Rare Disease Day
- NORD Debuts Rare Disease Day 2020 Video
- Head of the Herd: Bonnie Royster, CdLS Foundation
- Essay on protecting the Orphan Drug Act by NORD’s Peter L. Saltonstall published in CQ Researcher
- Head of the Herd: Mike Morris, TANGO2 Research Foundation
- Students for Rare Featured Student: Allison Herrity
- NORD Launches New Patient Assistance Program Providing Support to Eligible NMOSD Patients
- NORD Reaches 300 Members!
- Head of the Herd: Leah Schust Myers, FamilieSCN2A Foundation
- NORD Survey to Better Understand Rare Disease Diagnosis, Care and Treatment in the US: Take Part in Our 30 Year Follow-up
- #NORDinthenews: Washington Post profiles NORD’s Rare Disease Database and Rarediseases.org
- NORD’s Work with FDA Gives Patients a Chance to Be Heard in Drug Development
- What Questions Would You Ask About Gene Editing?
- A First Time for Everything: Marissa’s Running for Rare Story
- Rare Caregivers Corner: Debra’s Story
- #NORDintheNews: NORD Featured in Syndicated Article on Parents’ Intuition and Rare Disease
- NORD Survey to Better Understand Rare Disease Diagnosis, Care and Treatment in the US: Take Part in Our 30 Year Follow-up
- Reflections of a First Time NORD Summit Attendee: Matthew’s Story
- November is National Family Caregivers Month
- NORD supports Dr. Stephen Hahn’s nomination for FDA commissioner
- New NORD Member PALS Shares About the Impact of Free Flights for the Rare Disease Community
- NORD’s Rare Action Network Mobilized for a Successful Hill Day
- 2019 NORD Summit Breaks Records with Largest Attendance Ever!
- NORD Posts New Addition to Video Library – Pediatric Movement Disorders: Guidance for Parents & Physicians
- Information on current NATPARA® shortage
- A Joint Op-Ed from NORD and Friends of Cancer Research: Despite Criticism, FDA is Doing What is Right By Rare Cancer Patients
- Voices of Rare Cancer: Lisa’s Story
- Voices of Rare Cancer: Brian’s Story
- Voices of Rare Cancer: Susan’s Story
- Voices of Rare Cancer: McKinnon’s Story
- Voices of Rare Cancer: Aidan’s Story
- Voices of Rare Cancer: Lauren’s Story
- Today is #RareCancerDay!
- Amish Community Testimony
- Voices of Rare Cancer: Nicole’s story
- BioNews Service Previews 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit
- 2020 Living Rare, Living Stronger NORD Patient and Family Forum Location Announced
- Voices of Rare Cancer: Graeleigh’s Story
- NORD Expands Rare Disease Video Library
- Voices of Rare Cancer: Paul’s Story
- Students for Rare Featured Student: Jaime Tan
- Voices of Rare Cancer: Kristin’s Story
- NORD’s Mary Dunkle Comments on Diagnosis Challenges and Urges Caution on Using Internet Sources in New Article
- FDA Acting Commissioner to Deliver NORD Rare Summit Keynote
- NORD Announces Four New Board Members
- New Video on PTLD Added to NORD Rare Disease Video Library
- Registration Open for 2020 NORD Rare Summit
- Help for rare disease patients impacted by Hurricane Dorian
- NORD Awards Ten New Grants in Eight Disease States for Rare Disease Research
- The NORD Rare Disease Video Library Offers New Educational Resources
- Catch Living Rare Forum Plenaries and The Rare Impact Awards on Facebook Live!
- Exciting Rare Impact Awards Announcements
- The Platelet Disorder Support Association (PDSA): 2019 Abbey S. Meyers Leadership Award
- The Honorable Michael McCaul: 2019 Rare Impact Award Honoree
- The Honorable Sarah Davis: 2019 Rare Impact Award Honoree
- Deborah Skolaski: 2019 Rare Impact Award Honoree
- Madison Shaw: 2019 Rare Impact Award Honoree
- Dr. Debra Regier: 2019 Rare Impact Award Honoree
- Dr. Wendy Chung: 2019 Rare Impact Award Honoree
- Donna Appell, R.N.: 2019 Rare Impact Award Honoree
- Become a rare disease mythbuster during NORD’s Living Rare, Living Stronger Patient & Family Forum
- NORD Publishes Report on Primary Distal Renal Tubular Acidosis
- Living Rare, Living Stronger full agenda with speakers now available
- NORD Video Provides Advice for the Newly Diagnosed
- NORD announces expansion of three Patient Assistance Programs
- Orphan Drug Act Resolution Introduced in the House of Representatives
- Rare Disease Day 2019 Notes and Successes
- Connor’s Story in Honor of Rare Disease Day
- India’s Story in Honor of Rare Disease Day
- McCarthy Story in Honor of Rare Disease Day
- Brazeau Family Story for Rare Disease Day
- 10 Ways to Show Your Stripes this Rare Disease Day!
- Featured Student: Scarlett Eagle
- NORD Welcomes Rachel Sher as Vice President of Regulatory and Government Affairs, Lesli Nordstrom as Director of Marketing and Communications
- NORD IAMRARE KAT6A Patient Registry Launched
- Rare Disease Advocates Share Ideas for How to Mark Rare Disease Day 2019
- Rare Disease Day 2019 is Only 10 Weeks Away!
- New Study Finds Orphan Drug Exclusivity Working As Intended, On-Market Orphan Drug Prices Rise Slower than Common Drugs
- The 2019 Rare Impact Awards will take off at Space Center Houston, Saturday, June 22!
- Year-End Message from NORD’s President and CEO
- Orphan Drug Act Resolution Introduced in Congress
- NORD Creates Rare Disease Patient & Caregiver Resource Center
- Chat, Chew, and Be Challenged! Lunchtime Learning at the NORD Rare Summit
- Three Big Reasons Not to Miss the 2018 NORD Rare Summit
- NORD 35th Anniversary Blog Series: 2010-2018 (Present)
- Patient/Caregiver Opening Address Announced for NORD Rare Summit
- Join us at the 2018 NORD Rare Summit
- NORD Issues Statement on the Passing of Dr. Robert M. Campbell
- FDA Commissioner Dr. Scott Gottlieb Confirmed as 2018 NORD Rare Summit Keynote Speaker
- Get to know Jill Pollander, NORD’s new Director of Patient Services
- Spencer and Levy-Yeboa Syndrome, the Story of Our Son
- NORD 35th Anniversary Blog Series: 1990s-2000s
- Last Rare Disease Day Event of 2018 a Success in Pennsylania
- NORD Meets with HHS Secretary Azar to Discuss Needs of Rare Disease Patients
- NORD Issues Statement on Increasing Use of CoPay Accumulator Programs in Health Plans
- NORD Issues Statement Regarding House Passage of the Right to Try Act (S.204)
- Save the Date for NORD’s 2018 Rare Summit
- NORD statement in response to American Patients First, the Trump Administration Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs
- Watch LIVE: NORD’s 35th Anniversary & Rare Impact Awards Celebration
- NORD 35th Anniversary Blog Series: The Early Years (1980s)
- 35 Ways and Growing: NORD’s Service to the Rare Disease Community
- Robert Campbell, Jr., M.D.: 2018 Lifetime Achievement Award Honoree
- Estelle Benson, Founder of GBS|CIDP Foundation International: Abbey S. Meyers Leadership Award Honoree
- Richard Pazdur, M.D.: 2018 Public Health Leadership Award Honoree
- Patrick Dunegan: 2018 Rare Impact Award Honoree
- Elisabeth Dykens, Ph.D.: 2018 Rare Impact Award Honoree
- Maria Kefalas, Ph.D.: 2018 Rare Impact Award Honoree
- Ellen V. Sigal, Ph.D.: 2018 Rare Impact Award Honoree
- Chris Ulmer: 2018 Rare Impact Award Honoree
- NORD Shares News on 35th Anniversary Celebration Presenting the Rare Impact Awards
- NORD Issues Statement Regarding Governor Bevin’s Veto of Kentucky Senate Bill 7
- NORD Reaction to Recently-Enacted Government Spending Bill
- NORD Issues Statement Regarding House Vote on Right to Try
- National Organization for Rare Disorders (NORD) Announces Honorees for 2018 Rare Impact Awards
- NORD Joins Over 100 Patient and Provider Organizations in Opposition to Right to Try
- NORD Issues Statement Regarding House Vote on Right to Try
- 83 Patient and Provider Organizations Remain Opposed to Right to Try Legislation
- Another Successful Rare Disease Day®
- NORD Joins 125 Patient Organizations in Support of Medicaid Formulary Access
- One-pager from NORD on Opposition to Right to Try Act
- NORD Statement Regarding State Proposals to Alter Their Medicaid Program
- Current Funding Opportunity: NORD Research Grants for Rare Diseases
- NORD Sends Letter to House of Representatives on Right to Try Act
- NORD Issues Statement on Confirmation of New Secretary of HHS, Alex Azar
- Letter from NORD to Majority Leader Mitch McConnell and Minority Leader Chuck Schumer in support of Alex Azar’s candidacy
- NORD Issues Statement in Response to Senator Orrin Hatch’s Announced Retirement
- NORD Awards Five New Grants for Rare Disease Research
- NORD Issues Statement in Response to Congressional Consideration of the Tax Cuts and Jobs Act Conference Report
- Together, we can be stronger for those that need us.
- Over 160 Patient Organizations Reemphasize Support for the Orphan Drug Tax Credit
- NORD Issues Statement in Response to Senate Vote on the Orphan Drug Tax Credit
- NORD Publishes Information for Physicians and Patients on Three Rare Diseases
- Statement by 91 Patient Organizations in Opposition to the House of Representatives’ Repeal of the Orphan Drug Tax Credit
- Statement by 36 Patient Organizations in Opposition to Senate’s Proposed Weakening of the Orphan Drug Tax Credit
- NORD Publishes New Educational Resource on Li-Fraumeni Syndrome
- NORD Issues Statement on Nomination of Alex Azar for Secretary of the Department of Health and Human Services
- NORD Issues Statement in Response to Senate Finance Committee’s Proposal to Weaken the Orphan Drug Tax Credit
- NORD Issues Statement in Response to Proposed Repeal of the Orphan Drug Tax Credit (ODTC)
- NORD Issues Statement on Senate Leadership’s Recent Update on “Graham-Cassidy” Proposal
- FDA Announces First Gene Therapy in U.S.
- Downloadable Hurricane Harvey Emergency Relief Resources
- NORD Issues Statement in Response to the Signing by President Trump of the Food and Drug Administration Reauthorization Act of 2017
- Advance the Dialogue at the NORD Rare Summit
- NORD Issues Statement on the Latest ACA Repeal and Replace Developments
- NORD Publishes New Rare Disease Report on Retinal Vasculopathy with Cerebral Leukodystrophy (RVCL)
- The BCRA and Healthcare Reform: NORD’s Latest Update
- NORD Announces Keynote Speakers for 2017 Rare Diseases and Orphan Products Breakthrough Summit
- NORD Issues Statement Regarding the Release of Revised Draft of the Better Care Reconciliation Act
- The National Health Council and NORD Commend House Passage of FDA User Fee Reauthorization; Urge Senate to Follow Suit
- NORD Publishes New Rare Disease Report on Bosma arhinia microphthalmia (BAM) syndrome
- NORD Rare Action Network℠ Releases Summer 2017 State Policy Legislative Tracker
- NORD Issues Statement on the FDA’s Orphan Drug Modernization Plan
- NORD Issues Statement in Response to Delay in Consideration of the Senate Better Care Reconciliation Act
- NORD Leads Rare Disease Community Days of Action Campaign to Protect Healthcare Coverage
- NORD Publishes Report on Hereditary Sensory Autonomic Neuropathy Type 1E
- NORD Issues Statement on Confirmation of New FDA Commissioner, Dr. Scott Gottlieb
- Sign-on letter from 120 patient organizations urging Congress to reauthorize FDA User Fee Agreements
- NORD Issues Statement on Today’s Passage of the American Health Care Act
- Cynthia Tifft, M.D., Ph.D – 2017 Rare Impact Award Honoree
- Christine Grube – 2017 Rare Impact Award Honoree (Awarded Posthumously)
- Dr. Robert J. Desnick, MD, Ph.D – 2017 Rare Impact Award Honoree
- Rare Disease Summer Family Camp: A Place to Call Home
- Join NORD and ABC News for a Rare Disease Day® Tweetchat on Feb. 28
- Raise Your Hand for Rare Disease Research
- Family Moves Across State Lines to Receive Better Access
- A Message from Our President and CEO
- Happy Father’s Day from NORD
- NORD Members Share Great Resources for ER Visits During #RareERChat
- NORD Summit 2016
- Watch Live: Tomorrow’s Rare Impact Awards Ceremony
- Desiree Lyon Howe: 2016 Rare Impact Award Honoree
- Dr. Stephen Cederbaum: 2016 Rare Impact Award Honoree
- Noah Victoria: 2016 Rare Impact Award Honoree
- New Paper Addresses Health Care Costs and Patient Assistance Programs
- RareAction Network℠ Releases April State Legislative Tracker
- A Special Thank You to Volunteers in the Rare Disease Community
- Introducing the 2016 Rare Disease Day® Highlights Video
- NORD Founder Publishes Memoir Detailing Her Tireless Work as Parent-Advocate
- Neurology Reviews Publishes The Rare Neurological Diseaes Special Report
- Today is Rare Disease Day®
- Running on Air for Rare Disease Day
- Rare Disease Day Video for 2016 Launched
- Join the Running for Rare Team!
- FDA Invites Patient Organizations to Organize Their Own Meetings on the PFDD Model
- Register for the 2016 Rare Impact Awards
- Cure HHT Foundation
- CCHS Family Network
- Be a Hero: Help NORD Empower the Rare Disease Community in Washington D.C. and in all 50 States.
- Pachyonychia Congenita Project
- Be a Hero-Help NORD Help Patients Obtain Life-Saving Treatments
- Be a Hero: Help NORD Educate Current and Future Medical Professionals
- Joshua Frase Foundation
- National Brain Tumor Society
- Cutis Laxa Internationale
- CMTC-OVM
- Giving Spotlight: Parents Thank NORD for Saving Daughter’s Life
- The Guthy-Jackson Charitable Foundation
- Giving Spotlight: HomeServe USA Leadership and Employees Donate More Than $26,000 to NORD
- Taking on NYC and a Rare Disease: An Introduction
- Neuroendocrine Tumor Research Foundation
- NORD Releases First-Ever State Progress Report for Rare Diseases
- Rett Syndrome Research Trust
- Liam Cruz’ Diagnostic Journey
- Target Cancer Foundation
- Raise Your Voice: Rare Disease Day 2016 is Coming
- Narcolepsy Network
- The Undiagnosed: Orphans of the Rare Disease Community
- Dravet Syndrome Foundation
- Jill Zeigler’s Story: Life with aHUS
- HLRCC Family Alliance
- Rare Disease Day 2016 Theme and Slogan Announced
- NORD Named Charity of the Week By The Week Magazine
- The LAM Foundation
- Miss Pre-Teen Connecticut Visits NORD
- The Sturge-Weber Foundation
- Hope for Kids with Heart Disease: September is Children’s Cardiomyopathy Awareness Month!
- NORD’S Free Student Membership: Sign up Today!
- Miss Connecticut Talks to NORD about Rare Diseases, the Miss America Pageant, and Voting for America’s Choice
- Member Spotlight: FIRST
- New NORD PSA
- Rare Action™ Road Tour, Salt Lake City, UT
- Rare Disease Report and NORD Announce Editorial Collaboration
- Rare Action™ Road Tour, Salt Lake City, UT
- Rare Action™ Road Tour, Denver, CO
- Taking Rare Action™ on the Road
- Member Spotlight: CLOVES Syndrome Community
- Rare Action™ Road Tour, Oklahoma City, OK
- Miss Iron County Supports Rare Disease Advocacy and Awareness
- Rare Action™ Road Tour, Houston, TX
- Rare Action™ Road Tour, Tallahassee, FL
- Member Spotlight: LGDA
- Rare Action™ Road Tour, Atlanta, GA
- Q & A with Russ Illes, Author of Duct Tape Won’t Fix This
- 2015 Rare Disease and Orphan Products Breakthrough Summit
- Lauren Hill Dies at 19 after Battle with Brain Cancer, DIPG
- PMPRF President’s Statement on Stuart Scott’s Passing, and his Impact on the Appendix Cancer Community
- The Will to Live and the Strength for a Cure
- Life is a Special Occasion
- Multiple Endocrine Neoplasia Type 1: One Family’s Fight
- Taking Action for ACD
- NORD Celebration Highlights the Courage of Children and Adults Living with Rare Diseases
- Portraits of Courage Celebration
- NORD Launches Rare Action Network
- Multiple Endocrine Neoplasia Type 1: One Family’s Fight
- Taking Action for ACD
- Medical Research Should Be Everyone’s Priority
- Living with Blood Cancer
- Children’s Cardiomyopathy Awareness Month Urges Families to #KnowYourHeart this September
- The Will to Live and the Strength for a Cure
- NORD at Capitol Hill Hearing: Patients Have Unique Perspective That Needs to be Heard
- NORD Recommendations for Advancing Drug Discovery, Development, and Delivery
- Are Civility and Mature Behavior Really Too Much to Expect?
- Making Sure The Rare Disease Patient Voice is Heard on Capitol Hill
- NORD Calls Proposed Repeal of Orphan Drug Tax Credit “Anti-Patient and Anti-Public Health”
- FDA Creates New Web-Based Educational Tool About the Agency
- Our Shared Vision for Rare Disease Day
- BioNJ Presents Patient Advocate Award to NORD
- Building on Policy Momentum
- Statement on Congressman Waxman’s Retirement
- Genzyme and NORD Establish Program to Help Undiagnosed Patients with Rare Diseases
- Staying Focused on What Really Matters About the ACA
- 30 Years After the Orphan Drug Act: It Still Takes Too Long for People with Rare Diseases to Get an Accurate Diagnosis
- Even “Well Known” Rare Diseases Pose Diagnosis Problems
- Enrollment in Your Health Insurance Marketplace
- Supreme Court Decision Consistent With Longstanding NORD Position
- Orphan Drug Act at 30: Will Success Become Too Expensive?
- NORD Issues Statement on the Death of Actor Jack Klugman
- Patients Will Benefit From New Public-Private Partnership
- What Would You Consider “Essential Health Benefits” For the Disease of Concern to You?
- The Story Behind the Rare Disease Day School Curriculum
- Glybera Becomes First-ever Gene Therapy Approved in Europe
- Category: Medical
- Featured Student: Jennifer Shoskes
- NORD Seeks Proposals for CME Partnership
- Early Diagnosis: Perspectives from NORD Student Chapter Leader, Harjot Randhawa
- NORD Publishes New Report on RYR-1 Related Diseases
- Beth Nguyen, R.N. – 2017 Rare Impact Award Honoree
- Matthew Might, Ph.D. – 2017 Rare Impact Award Honoree
- Ellie McGinn – 2017 Rare Impact Award Honoree
- Dr. Frederick Kaplan, M.D. – 2017 Rare Impact Award Honoree
- Key Dates Approaching for NORD Summit
- NORD Hosts Tweetchat with Texas Children’s Hospital on Navigating the Pediatric ER (May 26)
- New SIDS Study Says Other Factors May Increase Risk
- NORD Publishes Guide to Promote Physician Awareness of Rare Lung Disorder
- Rare Disease of the Day
- Saving Eliza
- Thoughtful Biosimilars Policy Is Best Way to Ensure Optimum Patient Outcomes
- Images from the Portraits of Courage Celebration
- Engineering Treatments for Rare Disorders
- A CRISPR Approach to Genome Editing
- Informed Consent in Biobank Research
- New Guidelines on Testing Kids’ DNA–the Cliff’s Notes Version
- Can IBM Watson Replace Dr. Dhaliwal? Probably Not!
- Creative Collaborations May Be Key to Reducing Chemotherapy Risks
- Category: Patients & Members
- Tara O’s Story in Honor of Rare Disease Day
- Ramona’s Story in Honor of Rare Disease Day
- Tara Z’s Story in Honor of Rare Disease Day
- Hudson’s Story in Honor of Rare Disease Day
- Rare Disease Day 2020 and the Mission I Choose to Accept, by Debbie Drell, Director of Membership for NORD
- NORD Announces Sing Me a Story Raffle for Upcoming Living Rare, Living Stronger Patient and Family Forum
- Why Rare Disease Day Is Important to Me, by Chris Madden
- Gina and Gia’s Story in Honor of Rare Disease Day
- Wyatt’s Story in Honor of Rare Disease Day
- Zuhriah’s Story in Honor of Rare Disease Day
- Tucker’s Story in Honor of Rare Disease Day
- Rare Caregivers Corner: Q&A with Stephanie
- Rare Caregivers Corner: Cathy’s Respite Story
- Upcoming Externally-Led Patient Focused Drug Development Meeting for PKD Community
- NORD’s 2019 Living Rare, Living Stronger Weekend Highlights
- Arthur’s CIDP Story
- Hear from NORD’s registry community
- Fibrolamella Cancer Foundation Thanks NORD for Help with SSA Compassionate Allowance Listing
- Recap: NIH NCATS Day – Partnering with Patients for Smarter Science at the NIH
- A Group No One Applies to Join
- Penn State Graduate Rare Disorder Survey
- Celebrating Awareness: One-Year Anniversary of Rare Disease of the Day
- September is Children’s Cardiomyopathy Awareness Month
- NORD Publishes Expanded Access FAQ
- Guest Blog: Tour For A Cure
- Advocacy Alert: Illinois Rare Disease Commission Bill
- RareAction Network℠ Releases May/June State Policy Legislative Tracker
- Register for a FREE Webinar on the Undiagnosed Diseases Network (UDN)
- Advocacy Alert: Illinois Edges Closer to Establishing Rare Disease Commission
- Apply Now: The Hole in the Wall Gang Camp Hosts First-Ever Rare Disease Family Camp
- Notification (2/25/2016): Patient Assistance Programs
- American Plasma Users Coalition (A-PLUS) Applauds Recent Move by Department of Health and Human Services to Limit Out-of-Pocket Costs
- Rare Disease Artists’ Work Traveling Globe to Raise Awareness
- NORD and University of Maryland Partner on PCORI Award to Advance Rare Disease Research
- CMS Turns 50!
- Illinois Man Uses His Myelofibrosis Story to Help Others
- Knowing More About Noonan
- Living With Essential Thrombocythemia (ET)
- Not a Dry Eye in the Room: Families Tell FDA about Their Daily Struggles
- VHL Alliance and NORD Launch New International Databank
- Trivia Can Save Lives: How a Jeopardy Question Raised Awareness of a Rare Disease
- Adventures of an Advocate: Annette De Bow’s Big Journeys
- The Power of Words
- What are Your Favorite Rare Disease Movies?
- High-School Students Raise Awareness
- Privacy Protection in Whole Genome Sequencing
- Campaign to Educate State Legislators for Rare Disease Day
- Barretstown – a Serious Fun Camp!
- Category: Research
- Giving Spotlight: Honoring Mom-Mom’s Legacy with Research and Awareness
- NORD Announces Research Grants Available for Study of Rare Diseases
- NORD Awards New Research Grants for Rare Disease Research
- Research Grants for Rare Disease Research Available from National Organization for Rare Disorders (NORD)
- Registries for Rare Diseases: Involve the Patient
- Category: Advocacy
- Bridget’s Story in honor of Rare Disease Day
- Tik Tok Influencer Mark Anastasio Challenges Connecticut Students to Stripe Out Their Schools
- NORD Issues Statement on FDA’s Impending Expanded Access Call Center
- NORD Invites Video Submissions on Advocacy Experience
- NORD Responds to the Administration’s “Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs”
- NORD Issues Statement on Recent CMS Actions Pertaining to Patient Access Within Medicaid Formularies
- TAKE ACTION: Join NORD & Over 100 Patient Organizations in Supporting the RARE Act
- Your Help is Needed to #SaveCHIP
- Join Us for Rare Disease Day®
- Join NORD & SSA in Forum on Compassionate Allowances & Rare Diseases
- Protecting Your Healthcare Coverage
- Over 140 Patient Organizations Support the Orphan Drug Tax Credit
- Rare Disease Advisory Council Bill Moves through Pennsylvania House
- Advocacy Alert: Tell Congress to Act Now and Pass FDA User Fee Agreements
- NORD Rare Action Network℠ Releases Spring 2017 State Policy Legislative Tracker
- Brandon Hudgins – 2017 Rare Impact Award Honoree
- Reflections on FDA Listening Session
- Expansion of Medical Foods Coverage for Military Families Heads to the President, Having Passed in the Senate
- House Votes to Expand Medical Foods Coverage for Military Families
- House to Vote on New Version of 21st Century Cures Act Tomorrow
- November 15th Day of Action: 21st Century Cures
- $1.1 Billion in Federal Funding to Fight the Zika Virus
- The Hill publishes NORD op-ed: Congress must act now to help 15 million sick children
- ICER Comments Submitted
- NORD Responds to AHIP Report on Orphan Drug Act
- NORD Issues Statement in Response to PDUFA VI Commitment Letter
- NORD Issues Statement as Senate Postpones Vote on Cures Legislation
- July 13 Day of Action: Last Chance for Zika Funding
- Register: Rally for Medical Research Hill Day 2016, Sept. 21-22
- Rare Action Network: NORD and Patient Groups in Oregon Seek to Reduce Out-of-Pocket Drug Costs for Patients
- Today, Tell Congress We Need #CuresNow
- Wall Street Journal Publishes NORD Letter to the Editor
- Help Tara Pass School Bus Legislation
- What the Omnibus Bill Means for Rare Diseases
- National Spotlight on FDA and Duchenne Muscular Dystrophy: What This Means for Everyone with a Rare Disease
- NORD Supports Nomination of Dr. Califf for FDA Commissioner
- NORD Issues Statement Applauding the Approval of Ensuring Access to Clinical Trials Act
- NORD Issues Statement on FDA Rejection of Citizen Petition
- Bennet, Burr, Warren, Hatch Introduce Bill to Help Patients by Facilitating Faster Development of Drugs for Rare Diseases
- FDA: Another tool helping developers navigate the difficult road to approval of drugs for rare diseases
- Urge Your U.S. Senators to Attend Sept. 16th Briefing on Neurological Disease Research
- Advocating for Rare Disease Patients at the NIH
- NORD Signs Letter with Center for Responsible Science Calling for Changes at the FDA
- NORD Files Amicus Brief to Protect Drug Development Incentives in the Orphan Drug Act
- Ensuring Access to Clinical Trials Act of 2015 Passes in the U.S. Senate
- NORD Issues Statement on House’s Approval of 21st Century Cures Initiative
- NORD Submits Final Comments on 21st Century Cures Act
- FDA Public Meeting on Chagas Disease Patient-Focused Drug Development
- NORD Outlines Steps to Promote Innovative Drug Discovery, Development and Delivery for Americans with Rare Diseases
- NORD Board Member Testifies Before Energy & Commerce Health Subcommittee
- Proposed Rules for Health Insurance Market Reforms and Essential Health Benefits
- Category: Industry
- Category: Sticky Posts for Homepage
- NORD’s Educational Support Program
- NORD State Report Card
- COVID-19 Vaccines Update
- Rare Disease Day Merch Now Available
- Krabbe Drug Development Meeting
- Interested in Forming a Foundation?
- Krabbe Virtual Meeting
- NORD Summit 2020
- NORDpod
- Visit the NORD Store
- COVID-19 Rapid Response Leadership Series
- COVID-19 Community Survey Report
- Living Rare, Living Stronger Forum
- NORD COVID-19 Relief Programs
- NORD Guidance on Coronavirus Prevention and Risk
- Support NORD’s COVID-19 Programs
- Head of the Herd
- Caregiver Respite Program
- Join NORD on Rare Disease Day
- NORD Patient Assistance Program for 2020
- Living Rare Living Stronger
- Category: Sticky Posts for Patient Organizations
- Category: Sticky Posts for Patients and Families
- Category: Sticky Posts for Industry
- Category: Sticky Posts for Clinicians and Researchers
- Category: Sticky Posts for Advocate
- Category: Events
- A Celebration of NORD’s 35th Anniversary
- Nominations for the Rare Impact Awards Close Friday, January 12
- FDA Commissioner Dr. Robert Califf Delivers Keynote Address at NORD Summit
- Running for Rare Named an Official Charity Partner
- See What’s New and Exciting at the NORD 2016 Summit, Oct. 17-18!
- Join the Team: Running for Rare Takes on Hartford
- NORD Webinar Series
- Rare Disease Day
- Category: Patient Stories
- Amy’s Story in Honor of Rare Disease Day
- Ashlee’s Story in Honor of Rare Disease Day
- Bradley’s Story in Honor of Rare Disease Day
- Olivia’s Story in Honor of Rare Disease Day
- Tristan’s Story in Honor of Rare Disease Day
- Rare Caregiver Respite: Beth’s Story
- Kayte & Ashley’s Gastroschisis Story
- Nicole’s Gastroschisis Story
- Rare Caregiver Respite: Leah’s Story
- Rare Caregiver Respite: Debra’s Story
- Jenny’s ACPMP Story
- Ana’s Story for Rare Disease Day
- Mackenzie’s Mission
- Becoming a Marathon Runner
- Living with ALPS
- Tougher than an NFL Football Player
- Living Bella Soul
- Life with CGD
- Living with Seizure Disorder
- They Said things like, “She’ll Grow Out of It”
- From Mother to Daughter
- Giving Spotlight: Elizabeth and Chris Honor Son, Obie, with Donations to NORD, a Favorite Charity
- Giving Spotlight: Paula Mann Honors Son, Garrett, with Donation to NORD
- Guest Blog: A Parent Advocate & Author Discusses Courage
- The Fight for a Cure for Duchenne
- Category: Get Involved
- Category: Rare Disease Day
- Category: Head of the Herd
- Category: COVID-19
- Category: #NORDintheNews
- Category: RareLaunch
- Category: RDCA-DAP
- Category: Newborn Screening
- Category: Voices of Rare Cancer 2020
- Category: RareInsights
- Category: RDACs
- Category: Students for Rare
- Category: State Report Card
- Category: Medicaid
States
- Washington
- Virginia
- Vermont
- Utah
- Texas
- Tennessee
- South Dakota
- South Carolina
- Wyoming
- Wisconsin
- West Virginia
- Rhode Island
- Pennsylvania
- Missouri
- Mississippi
- Minnesota
- Michigan
- Oregon
- Oklahoma
- North Dakota
- North Carolina
- New York
- New Mexico
- New Jersey
- New Hampshire
- Nevada
- Nebraska
- Montana
- Ohio
- Massachusetts
- Maryland
- Maine
- Louisiana
- Kentucky
- Kansas
- Iowa
- Indiana
- Illinois
- Idaho
- Florida
- California
- Arkansas
- Alaska
- Georgia
- Arizona
- Hawaii
- District of Columbia
- Delaware
- Connecticut
- Colorado
- Alabama
Policy Issues
- Medicaid Financial Eligibility
- Rare Disease Advisory Councils
- Protecting Patients in State-Regulated Insurance
- Protecting Patients in State Medicaid Programs
- Prescription Drug Out-of-Pocket Costs
- Step Therapy (Fail First)
- Medical Nutrition
- Newborn Screening
Patient Stories
- Tammy Jones, Arkansas Rare Action Network Volunteer Ambassador
- Pat Geurds, New Jersey Rare Action Network Volunteer Community Engagement Liaison
- Nick Parent, Tennessee Rare Action Network Volunteer Community Engagement Liaison
- Mike Hu, California Rare Action Network Volunteer Ambassador
- Maria Bellefeuille, Illinois Rare Action Network Volunteer Ambassador
- Kenny Brooks, West Virginia Rare Action Network Volunteer Ambassado
- Jennifer Dunegan, Kentucky Rare Action Network Volunteer Ambassador
- Jana Monaco, Virginia Rare Action Network Volunteer Ambassador
- Erica Barnes, Minnesota Rare Action Network Volunteer Ambassador
- Danyelle Sun, Wisconsin Rare Action Network Volunteer Ambassador
- Charlene York, Ohio Rare Action Network Volunteer Ambassador
- Anne Rugari, Florida Rare Action Network Volunteer Ambassador
Venues
- https://zoom.us/j/99692687399?pwd=WTNjMzFNR2hPQVJ5MytjQUdBM0plUT09
- Zoom
- Zoom: https://ncsu.zoom.us/j/91469394854
- Tjhebelopele Primary School Bloemfontein Free State
- Twitch
- Virtual
- 36839
- 42167
- online
- Dyne Therapeutics
- The North Door
- Succasunna United Methodist Church
- Salem
- Triumph Brewing Company
- Crowne Plaza Dublin Airport
- One Police Plaza, NY, NY
- Biruni University
- Pair of Dice
- Pomona College Frank Blue Room, Frank Dining Hall 260 E. Bonita Avenue
- University of Florida
- Dexter, MI
- The Get Down
- Syneos Health Corporate Headquarters
- Chabot Space & Science Center
- Children’s Hospital Colorado
- Flatirons Community Church
- Andi's Coffee and Bakery
- Riverton Branch Library
- Hilton Los Angeles Airport
- Legislative Office Building, Room 104A
- Gardner Room at the Wehr Nature Center
- THIRUVANATHAPURAM
- St. Joseph’s Indian High School ( Besides Mallya Hospital)
- Carnegie Hall
- Sip ~N~ strokes
- Sip~N~Strokes
- children's museum Tucson
- Children's of Alabama Bradley Lecture Center
- Avani Atrium Bangkok Hotel
- Bangkok, Thailand
- University of Notre Dame
- Grant Sawyer State Office Building
- McNamara Alumni Center
- Oklahoma Science Museum
- Pollard Library
- The North Door
- Georgia State Capitol
- Omni Parker House
- Kentucky State Capitol
- New York State Capitol
- State House Inn
- The DoSeum (San Antonio Children’s Museum)
- McCarthy Pavillion
- Common Man Restaurant
- Tucson Children’s Museum
- New Hampshire State Capitol Building Cafeteria
- Staples Center
- Arkansas College of Osteopathic Medicine
- Sugar Land Town Square
- Connecticut State Legislative Office Building
- Colorado State Capitol Building, 2nd Floor West Foyer
- Sugar Land Memorial Park
- Harrington Discovery Institute University Hospitals
- Hospital for Special Care
- Prince Conference Center
- International Spy Museum
- White Oak Campus: The Great Room Conference Center
- White Oak Campus: The Great Room Conference Center
- Lago Conferences & Events Center
- Lago Conferences & Events Center
- North Bethesda Marriott Hotel & Conference Center
- DoubleTree by Hilton Hotel Bloomington
- Aloft Hotel Dallas Love Field
- Kansas City Marriott Country Club Plaza
- Courtyard by Marriott Nashville Downtown
- Hotel SOFIA Barcelona
- Warwick Hotel Rittenhouse Square
- Virginia Gateway Shopping Center
- Atlanta Marriott Alpharetta
- Gaylord National Harbor Hotel
- Florida's Forest
- Boston City Hall
- Times Square Manhattan
- Michigan League- Henderson Room
- Alexandria Center for Life Science
- Raytheon Amphitheater at Northeastern University
- The Rivers Edge
- Steamboat Grand Hotel
- NIH - Main Auditorium, Building 45
- Microsoft Global Headquarters
- United Nations Headquarters, New York
- Zablocki Pavilion
- Medical University of South Carolina, Conference Room
- Pennsylvania State Capitol
- World Trade Center Portland
- Ohio State House, Governor Thomas Worthington Center
- New Jersey State Museum, Main Room
- Minnesota State Capitol, Rotunda
- New Hampshire State Capitol, Cafeteria
- Radisson Hotel Lansing at the Capitol, Banquet room, 2nd floor
- Bernard W. Brown Community Center
- Kentucky State Capitol, Rotunda
- The Inn at 835
- Georgia State Capitol, South Wing
- Legislative Office Building, 2nd floor Atrium
- Washington State Campus, Spokane Campus, Room SAC20
- The Health Museum
- Mississippi State Capitol, West Wing
- Arizona State Capitol, Lawn
- Oklahoma State Capitol, 4th Floor Rotunda
- The Trylon Cinema
- Martin Murphy Middle School
- Yale University School of Medicine
- St. Joseph's Children Hospital
- Trinity Presbyterian Church Fellowship Hall
- Tokyo, Japan
- Memorial Union (The Sun Room)
- Holiday Inn Aurelia
- TopGolf, Scottsdale
- Rome, Italy
- Golden Tulip Berlin
- Berlin, Germany
- Embassy Suites by Hilton Charlotte Uptown
- Crowne Plaza Hotel Seattle Airport - Grand Ballroom
- Hilton Crystal City
- The Scripps Research Institute-Florida
- UCLA Center for Health Sciences
- Hilton Americas-Houston
- Texas Scottish Rite Hospital for Children
- Northern Kentucky Convention Center
- Channahon Central Park
- The Carolina Inn
- Embassy Suites O’Hare Rosemont
- Pittsburgh Marriott City Center
- Embassy Suites Minneapolis Airport
- Renaissance Newport Beach
- SHERATON CHARLOTTE HOTEL AND LE MERIDIEN CHARLOTTE
- SHERATON CHARLOTTE HOTEL AND LE MERIDIEN CHARLOTTE
- Hilton DFW Lakes Executive Conference Center
- San Diego Marriott Del Mar
- The WHITEHALL
- Feestzaal Oude Vismijn
- Courtyard by Marriott Hartford Cromwell
- Akin Gump Strauss Hauer & Feld LLP
- Royal College of Physicians
- Marriott Minneapolis City Center
- Hyatt Regency Aurora-Denver
- Virtual Demonstration
- The Westin Los Angeles Airport
- Crowne Plaza Providence-Warwick
- Hyatt Regency Indianapolis
- UT MD Anderson Cancer Center
- Renaissance Baltimore Harborplace Hotel
- Messe Wien Congress Center, Vienna, Austria
- Houston Zoo
- Frank H Netter MD School of Medicine-Quinnipiac University
- Mission Baby Conference Center at UCSF
- Center for Total Health
- Grand Hyatt
- Lake Calhoun
- THE POOL & THE GRILL Restaurants - Seagram Building
- Bethesda North Marriott Hotel
- Roswell Park Cancer Institute Gaylord-Cary Room
- Fairmont Rey Juan Carlos Barcelona
- Snowbird Ski and Summer Resort
- Nashville Airport Hotel
- Hyatt Regency, Newport Beach
- Casa Del Mar
- The New York Academy of Sciences
- Sheraton Grand at Wild Horse Pass
- New York Marriott East Side Hotel
- New York Marriott East Side
- Loews Chicago O'Hare Hotel
- University of California-Luskin Conference Center
- University of Rochester-Saunders Research Building-Helen Wood Hall Auditorium
- Hancock Wellness Center
- Texas Children's Hospital-Pavillion for Women
- The New York Academy of Sciences
- Renaissance Washington D.C. Hotel
- Georgetown University
- College Park Marriott Hotel & Conference Center
- Washington Marriott Wardman Park
- Magnolia Hotel Houston
- Vivint Smart Home Arena
- Astor Crowne Plaza Hotel
- Crowne Plaza Redondo Beach and Marina
- Manhattan Beach Marriott
- Hilton Columbus at Easton
- Embassy Suites by Hilton Nashville Airport
- Hole in the Wall Gang Camp
- Westlake Park
- Hilton Phoenix Airport
- Ronald Reagan Building and International Trade Center
- Hotel Edison
- The Four Seasons Hotel St. Louis
- Bahia Mar Fort Lauderdale Beach
- Stony Brook University
- Hyatt Regency Downtown
- Sheraton Inner Harbor Hotel
- Rhode Island Convention Center
- Sun 'n Lake Golf Club
- Mayo Clinic-Ashton Taylor Auditorium
- Marriott Wardman Park
- Casa Del Mar
- Hampton Inn Philadelphia Convention Center
- DoubleTree Resort by Hilton Hotel
- Mission Bay Golf Course
- New York Academy of Medicine
- Royal Sonesta Hotel
- University of Penn's Jordan Medical Center
- Riverside Park
- Faulty House at Columbia University
- Hotel Leusden (van der Valk)
- Fenway Park
- Salt Lake Marriott Downtown at Creek City
- Bristol Community College
- Center for Advanced Medicine-3rd Floor Farrell Conference Room #1
- Tampa Marriott Waterside Hotel & Marina
- Grand Hyatt Washington
- B Resort and Spa
- Reagan Building and International Trade Center
- Cherry Creek Dam Road
- Memorial Sloan Kettering-Rockefeller Research Laboratory-Room 116
- Unnamed Venue
- Victorian Gardens
- Sheraton Philadelphia University City Hotel
- Columbia University - Bard Hall
- Crowne Plaza Boston Newton - Charles Ballroom
- University of Utah Medical Center - Univesity Guest House, Douglas Ballroom
- University of Texas Southwestern Medical Center - T. Boone Pickens Auditorium
- Johns Hopkins Hospital-Sheikh Zayed Tower, Chevy Chase Auditorium
- Faculty House at Columbia University
- Hilton New Orleans Riverside
- Johns Hopkins Hospital - Sheikh Zayed Tower, Chevy Chase Auditorium
- Hilton Garden Inn Downtown Austin
- Holiday Inn Bayside
- Gaylord Palms Hotel
- Renaissance St. Louis Airport Hotel
- Unnamed Venue
- Devou Park
- Embassy Suites Cincinnati River Center
- Crystal Gateway Marriot
- Endicott Park
- Normandale Lake Bandshell
- BEST Conference Center-Rm 125
- RPB Auditorium-Jules Stein Eye Institute at UCLA
- Crowne Plaza Orlando Downtown
- Mount Sinai Hospital
- 3 West Club
- Doubletree Rochester
- CURRENT @ 59
- Sofitel San Francisco Bay
- Courtyard Columbus Downtown
- Hilton Baltimore BWI
- Doubletree by Hilton Hotel Atlanta
- Doubletree by Hilton
- Mandarin Oriental New York
- Sheraton New Orleans Hotel
- Uptown, Minneapolis Running Room
- Intercontinental Miami
- Cincinnati Marriott at Rivercenter
- Doubletree Bedford Glen Hotel
- University of Chicago
- Sheraton City Centre Hotel
- Embassy Suites Downtown/Old Market
- Newport Beach Marriott Hotel & Spa
- Hyatt Regency Jersey City Hotel
- Hyatt Regency Jersey City Hotel
- The Westin Westminster
- Central Park
- Minneapolis Marriott City Center
- Rochester Marriott Mayo Clinic
- Eaglewood Resort
- TBD
- Quassy Amusement Park
- TBD
- Renaissance Denver Downtown City Center Hotel
- Atlanta Airport Marriott
- Hyatt Regency Crystal City
- Rochester Municipal Park
- Hilton Durham-Trinity Ballroom
- Medical University of South Carolina-Drug Discovery Building-Room DD110
- Coon Creek Trap & Skeet Club
- Hilton Long Beach
- Texas Children's Hospital-Pavilion for Women, 4th floor Conference Center
- Cincinnati Marriott RiverCenter
- Children's Hospital of Richmond at VCU-Brook Road Campus Auditorium
- Renaissance Atlanta Waverly Hotel & Convention Center
- Walt Disney World Swan and Dolphin Resort
- Marriott Rivercenter
- Sheraton Salt Lake City Hotel
- Boston Marriott Newton
- Guastavino's
- Boston Marriott Cambridge Hotel
- Rosen Shingle Creek Resort
- Paris Marriott Rive Gauche Hotel & Conference Center
- Unnamed Venue
- Gaylord National Resort & Convention Center
- Burlington Marriott
- Hyatt Regency
- Pitié-Salpêtrière Hospital
- Wyndham Boston Beacon Hill
- Polk School
- West Palm Beach Marriott
- DoubleTree by Hilton San Antonio Airpot
- DoubleTree by Hilton San Dieo-Mission Valley
- Sheraton Imperial Hotel & Convention Center
- Fred Hutchinson Cancer Research Center
- DoubleTree by Hilton Cincinnati Airport Hotel
- Hilton Washington D.C./Rockville Hotel
- Hilton Rockville Hotel
- Stone Mountain
- Hole in the Wall Gang Camp
- Tennessee State Capitol
- Muse Paintbar
- Vermont State House Event Location
- Mississippi State House Event Location-Section A 1st Floor
- New Jersey Business and Industry Association (NJBIA)
- New Hampshire State House Event Location
- Cherberg Building Conference Room A
- Liberty Plaza
- DoubleTree by Hilton at Sea World
- Unnamed Venue
- Unnamed Venue
- Unnamed Venue
- Wisconsin State House
- Virginia State House
- Utah State House
- Pennsyvania State House
- Oregon State House
- Oklahoma State House
- Ohio State House
- New York State House
- Minnesota State House
- Michigan State House
- Maryland State House
- Maine State House
- Kansas State House
- Iowa State House
- Indiana State House Event
- Illinois State House
- Hawaii State House
- Connecticut State House
- Colorado State House
- California State House
- Alabama State House
- Unnamed Venue
- NORD Headquarters
- Marriott St. Louis Airport hotel
- Howard County Fairgrounds
- Renaissance Orlando at SeaWorld®-Oceans Ballroom
- The Warner Theatre
- Ruth and Tristram Colket, Jr. Translational Research Building
- XO Lounge
- Unnamed Venue
- Unnamed Venue
- San Diego Marriott Mission Valley
- Unnamed Venue
- South San Francisco Conference Center
- Andrew W. Mellon Auditorium
- The Burroughes
- Holiday Inn Toronto International Airport Location
- MD Anderson Cancer Center
- Robert H. Lurie Medical Research Center-Baldwin Auditorium
- Sylvester Comprehensive Cancer Center
- Heberman Conferece Center
- Capitol Hill
- Hotel Monaco
- The Park
- Cleveland Clinic
- DoubleTree Hilton Chicago-Magnificent Mile
- The Fairmont Dallas
- Oakdale Room at the Turf Valley Resort
- The Hilton St. Louis at the Ballpark Hotel
- Mission County Park
- Beaver Meadows Golf Club
- J.W. Marriott
- Cohasset Golf Club
- NYU College of Dentistry
- The Embassy Suites Omaha Downtown/Old Market
- FDA White Oak Campus
- Crystal Gateway Marriott
- Unnamed Venue
- InterContinental Toronto Centre
- Tampa Marriott Westshore
- Chicago Marriott O'Hare
- Westin Boston Waltham
- Hyatt Santa Clara
- Cleveland Airport Marriott
- Embassy Suites Washington, D.C. - at the Chevy Chase Pavilion
- Westchester Marriott
- JW Marriott Phoenix Desert Ridge Resort and Spa
- Omni Providence Hotel
- Charlotte Marriott South Park
- Marriott Downtown at Key Center
- McCormick & Schmick’s
- Sheraton Wild Horse Pass Resort & Spa
- The Westin Riverwalk
- Morton's Steakhouse Dallas
- Morton's Steakhouse Sacramento
- Battle House Renaissance
- Ruth's Chris Steakhouse
- Unnamed Venue
- Ocenaire Seafood Room
- Sonesta Hotel
- Morton's Steakhouse
- Teaneck Marriott at Glenpointe
- Le Meridien Atlanta Perimeter
- The Westin Galleria Dallas
- Anaheim Majestic Garden Hotel
- Omaha Magnolia Hotel
- The Westin Minneapolis
- The Carlton Hotel
- Omni William Penn Hotel
- The Brown Palace
- Hyatt Santa Clara
- Cleveland Airport Marriott
- Omni Hotels and Resorts Jacksonville
- Doubletree by Hilton at SeaTac
- InterContinental Mark Hopkins San Francisco
- Grand Hyatt Seattle
- Hilton Garden Inn Chicago Downtown/Magnificent Mi
- Hilton Orlando Lake Buena Vista
- Grand Hyatt Seattle
- Joyce Niblack Memorial Conference on Myeloproliferative Neoplasms
- NORD Winter Regional Membership Meeting
- NORD Spring Regional Membership Meeting
- Salzburg Congress Center
- Salzburg Congress Center
- National Building Museum
- 2015 Portraits of Courage Celebration
- 7th International Conference on Children's Bone Health
- Salzburg Congress Center
- Unnamed Venue
Organizers
- National Health Council
- NORD Wisconsin Rare Action Network
- Rare Disease Network
- NORD Utah Rare Action Network
- NORD Illinois Rare Action Network
- Arizona Rare Action Network
- Foundation for Sarcoidosis Research
- APBD Research Foundation
- CurePSP
- NORD Connecticut Rare Action Network
- NORD Georgia Rare Action Network
- NORD Tennessee Rare Action Network
- NORD Florida Rare Action Network
- NORD Maine Rare Action Network
- NORD Delaware Rare Action Network
- NORD Louisiana Rare Action Network
- NORD South Carolina Rare Action Network
- NORD Oklahoma Rare Action Network
- NORD Indiana Rare Action Network
- NORD Kansas Rare Action Network
- NORD Virginia Rare Action Network
- NORD North Caorlina Rare Action Network
- NORD West Virginia Rare Action Network
- NORD Ohio Rare Action Network
- NORD California Rare Action Network
- Incyte
- CURE
- HCU Network America
- Neuromuscular Disease Foundation
- Pheo Para Alliance
- International Waldenstrom's Macroglobulinemia Foundation
- Sickle Cell Disease Association of America
- GRIN2B Foundation
- Sara's Cure
- Cure DRPLA
- National Ataxia Foundation
- Usher Coalition
- Usher Coalition
- SSADHD
- SSADHD
- Smith-Kingsmore Syndrome Foundation
- Organic Acidemia Association (OAA)
- MitoAction
- VHL Alliance
- Moebius Syndrome Foundation
- International Sacral Agenesis Caudal Regression Association
- Guthy-Jackson Charitable Foundation
- United Leukodystrophy Foundation
- Syneos Health
- New Jersey Rare Action Network
- Cure VCP Disease
- Rocket Pharmaceuticals
- NORD Nevada Rare Action Network
- College of Pharmacy University of Minnesota
- Oklahoma Rare Action Network
- Massachusetts Rare Action Network
- Logan Greenberg
- Georgia Rare Action Network
- MassBio
- Kentucky Rare Action Network
- New York Rare Action Network
- Illinois Rare Action Network
- Be Fierce Foundation
- Ryland Grace Memorial
- Wisconsin Rare Action Network
- MECP2 Duplication Foundation
- NORD New Hampshire Rare Action Network
- NORD Arkansas Rare Action Network
- NORD Texas Rare Action Network
- NORD Colorado Rare Action Network
- NORD
- NORD
- NORD
- Israel Ministry of Health
- ICORD
- ICORD
- Sofia Sees Hope
- Rare Kids Network
- The International Advocate for Glycoprotein Storage Diseases
- Rocket Pharma
- RARE.
- Angioma Alliance
- Rare Diseases International
- Child Neurology Foundation
- Adrenal Insufficiency United
- AAIDA
- Glut1 Deficiency Foundation
- Reagan-Udall Foundation
- CureSMA
- Legg Calve Perthes Foundation
- Friedreich's Ataxia Research Alliance
- Dup15q Alliance
- PSP Association
- Cloves Syndrome Community
- Global CMTC-OVM US
- Organic Acidemia Association
- PTEN Hamartoma Tumor Syndrome Foundation
- United Leukodystrophy Foundation
- Lymphangiomatosis & Gorham’s Disease Alliance
- The Alagille Syndrome Alliance
- The Ehlers-Danlos Society
- The Morgan Leary Vaughan Fund
- EURORDIS
- NIH
- XLH Network
- Congenital Hyperinsulinism International
- MDS Foundation
- Terrapinn
- Cystinosis Research Network
- The New York Academy of Sciences
- Platelet Disorder Support Association
- The University of Rochester
- Children's Cardiomyopathy Foundation
- FDA
- The Myasthenia Gravis Foundation
- NTM Info & Research, Inc.
- Terrapinn Inc.
- PRISMS
- The American Porphyria Foundation
- Rare & Undiagnosed Diseases Network
- Myasthenia Gravis Foundation of America
- Dup15-Q Alliance
- The Myelodysplastic Syndromes Foundation, Inc.
- Hole in the Wall Gang Camp
- BORN A HERO
- The Marfan Foundation
- APS Type 1
- The Association for Frontotemporal Degeneration
- Hemophilia Federation of America
- International Waldenstrom's Macroglobulinemia Foundation
- Georgiana Marshen
- MPN Education Foundation
- Spinal CSF Leak Foundation
- National Spasmodic Dysphonia Association
- San Diego Sockers
- Nephcure Kidney International
- Alport Syndrome Foundation
- cmtcovm
- TargetCancer Foundation
- The Cholangiocarcinoma Foundation
- American Partnership for Eosinophilic Disorders
- Multiple System Atrophy Coalition
- National Leiomyosarcoma Foundation
- Pulmonary Hypertension Association
- National Organization for Rare Disorders
- Narcolepsy Network
- Medical Device Innovation Consortium
- Multiple System Atrophy
- Richardson Centre for Functional Foods and Nutraceuticals
- Daybreak Children's Rare Disease Fund
- Castleman Disease Collaborative Network
- Cutaneous Lymphoma Foundation
- The Myositis Association
- International Pemphigus and Pemphigoid Foundation
- Kennedy's Disease Association
- Batten Disease Support and Research Asociation
- NBIA Disorders Association
- Myotonic Dystrophy Foundation
- Soft Bones
- Vasculitis Foundation
- Wilson Disease Association
- AMEN Support
- Hereditary Neuropathy Foundation
- Melorheostosis Association
- MPN Coalition
- American Syringomyelia & Chiari Alliance Project
- CMTC-OVM
- RYR-1 Foundation
- Chordoma Foundation
- United Mitochondrial Disease Foundation
- Myocarditis Foundation
- Scleroderma Foundation
- Chloe's Fight for Rare Diseases
- Alpha-1 Foundation
- LAM Foundation
- Association of Gastrointestinal Motility Disorders (AGMD)
- The Transverse Myelitis Association
- Alternating Hemiplegia of Childhood Foundation
- United Leukodystrophy Foundation
- The Oley Foundation
- CurePSP
- CurePSP
- Organic Acidemia Association
- Reflex Sympathetic Dystrophy Syndrome Association (RSDSA)
- Hydrocephalus Association
- K-T Support Group
- Rettsyndrome.org
- Turner Syndrome Society of U.S.
- American Brain Tumor Association
- American Behcet's Disease Association
- Pulmonary Fibrosis Foundation
- The Myelin Project
- Moebius Syndrome Foundation
- Bridge the Gap-SYNGAP ERF
- The LAM Foundation
- American Partnership for Eosinophilic Disorders (APFED)
- Galactosemia Foundation
- Osteogenesis Imperfecta Foundation
- National Fragile X Foundation
- Immune Deficiency Foundation
- Sarcoma Foundation of America
- The Chordoma Foundation
- National Tay-Sachs & Allied Diseases Association
- ML4 Foundation
- Consortium of Multiple Sclerosis Centers
- Cure HHT
- Turner Syndrome Society
- ECD Global Alliance
- VHL Alliance
- The Association for Frontotemporal Degeneration
- Aplastic Anemia & MDS International Foundation
- Worldwide Syringomyelia & Chiari Task Force
- Hole in the Wall Gang Camp
- Martha Staples-Running for Rare Runner
- Unnamed Organizer
- CdLS Foundation
- Unnamed Organizer
- National Niemann-Pick Disease Foundation
- Fat Disorders Research Society
- Danielle Kempe
- Carrie Lucas
- Unnamed Organizer
- The Children's Hospital of Philadelphia
- Joseph Filippello
- FIRST
- GBS CIDP Foundation International
- Canadian Organization for Rare Disorders
- Answering TTP
- A Cure in Sight
- Ocular Melanoma Foundation
- Unnamed Organizer
- Kimberly Anderson
- Unnamed Organizer
- KD Golf CNY
- PFF Patient Communication Center
- Daniel Hackett
- Unnamed Organizer
- Unnamed Organizer
- Barbara Kass
- Unnamed Organizer
- Taryn Byrne
- Angie Onofre
- Angie Onofre
- Angie Onofre
- Angie Onofre
- Angie Onofre
- Angie Onofre
- Angie Onofre
- Jennifer Price
- Lisa Schill
- NORD (Alexandra Freitas)
- NORD (Lauren Rosato)
- NORD (Allie Frietas)
- NORD (Kristen Angell)
- NORD (Susan Olivo)
- NORD
- NORD
- National Building Museum
- Salzburg Congress Center
Events
- Living Rare, Living Stronger NORD Patient and Family Forum
- 2021 Rare Impact Awards
- The Land of the Free, Home of the Brave, Family Conference
- Chemistry Conferences
- Chemistry Conferences 2021
- CurePSP Virtual Spring Family Conference
- 11th World Congress on Breast Cancer & Therapies
- 11th World Congress on Breast Cancer & Therapies
- VHL Alliance in NYC
- EDS ECHO: Pain Management - A European Perspective
- Aplastic Anemia & MDS International Foundation Spring Patient and Family Conference
- International Pemphigus and Pemphigoid Foundation Rocky Mountain Support Group
- World Pediatrics Conference
- World Neuroscience and Psychiatry Conference
- International Pemphigus and Pemphigoid Foundation South Florida Support Group
- CurePSP Cookie Decorating Fundraiser
- Creating Your Medical Advisory Board
- 64th NAF Annual Ataxia Conference
- Sergeant at Arms, Beaumont Toastmasters Brain Wise Saves Lives wisdom
- McGill RareDIG Rare Disease Day 2021
- International Pemphigus and Pemphigoid Foundation Southern California Support Group
- Virtual Genetic Pop-Up - hosted by Hannah
- Virtual Genetic Pop-Up - hosted by Emma
- Advocating for a Rare Disease Advisory Council in Your State
- New Jersey Rare Disease Day Virtual Event
- Webinar: COVID-19, Nutrition, and Meeting the Needs of the Most Vulnerable
- ROOTS: Jewish Genes and Cuisine
- Utah Rare Disease Day Virtual Event
- Patients: The Heroes of Rare Disease Research
- Maine Rare Disease Day Virtual Event
- Rare Disease Day at NIH
- Community Heroes Rare Disease Day Panel Discussion
- Ehlers-Danlos Support Group of Jacksonville Rare Disease Day Social
- Free To Be You
- Rare Disease Day 2021 With CENTOGENE - Virtual Conference and Film Festival
- Rare Disease Day 2021 With CENTOGENE – Virtual Conference and Film Festival
- Texas Rare Disease Day Virtual Event
- NYPD VIRTUAL RARE DISEASE DAY 2021
- Delaware Rare Disease Day Virtual Event
- University of Michigan Virtual Rare Disease Day Event
- World Rare Disease Day
- Virtual Run for Rare
- Raise Awareness for KLS in Times Square
- Rare Disease Day 2021
- Louisiana Rare Disease Day Virtual Event
- BioNews #WhatMakesMeRare Virtual Event
- Sarcoidosis Wellness Series: Effective and Empathetic Communication
- University of Michigan Virtual Rare Disease Day Event
- Phelan Lucky February Virtual Run
- Arkansas Rare Disease Day Virtual Event
- Rare Disease Day Symposium
- Rare Disease Day at Frank H. Netter School of Medicine
- Frank H. Netter School of Medicine Rare Disease Day Symposium
- Rare Disease Day Bay Area
- Healthcare and Advocacy Careers in Rare Diseases
- Guthy-Jackson Charitable Foundation Rare Disease Day Event
- Virtual Genetic Pop-Up - hosted by Jamie
- UCLA Rare Disease Day 2021!
- Georgia Rare Disease Day Virtual Event
- Webinar: Real World Data and the COVID-19 Pandemic
- Illinois Rare Disease Day Virtual Event
- Tennessee Rare Disease Day Virtual Event
- Minnesota Rare Disease Day Virtual Event
- Lunch and Learn at Atlanta Bread Woodstock
- Telehealth in Rare Disease Clinical Practice and Research: The Silver Lining Now and Beyond COVID-19
- University of Michigan Virtual Rare Disease Day Event
- University of Michigan Virtual Rare Disease Day Event
- 2021 Florida Virtual Rare Disease Day Online Event
- Florida Rare Disease Day Virtual Event
- Connecticut Rare Disease Day Virtual Event
- South Carolina Rare Disease Day Virtual Event
- New York Rare Disease Day Virtual Event
- Rare Disease Day with North Carolina State University (NCSU), University of North Carolina-Chapel Hill (UNC), the Wake Forest School of Medicine, and Obra D. Tompkins High School
- Advancing Health Equity: A Community Conversation for Rare Disease Day
- Oklahoma Rare Disease Day Virtual Event
- Wisconsin Virtual Rare Disease Day
- The Orphan Disease Center 10th Anniversary Virtual Symposium: A New Era of Rare Disease Diagnostics and Therapeutics
- Virtual Rare Disease Symposium
- RARE DISEASE DAY EVENT 2021: RARE COMMUNITY
- Innovation in Rare Disease: Making Progress with Cell & Gene Therapies
- Rare Disease Day 2021 at Vanessa Research
- Zebra 5K Virtual Run
- NORD Running for Rare Program
- Rare Liver Diseases Month
Carousels
CME Videos
- von Willebrand Disease Management for Hematologists and Referring Clinicians: Diagnosis, Treatment, and Emerging Strategies
- Molecular Profiling and Targeted Therapies Shifting the Paradigm for Cholangiocarcinoma (Bile Duct Cancer)
- Reducing Diagnostic Challenges and Expanding Treatment Options for Advanced GIST
- All About NETs: Disease Pathway, Treatment, and Clinical Trials for GEP-NETs, Pheochromocytoma, and Paragangliomas
- Where are we Headed in Thyroid Cancer? New and Emerging Treatment Approaches
- A Critical Update for the Multidisciplinary Team in Differentiating and Tailoring Management of Sarcomas
- Managing Relapsed/Refractory Hairy Cell Leukemia in the Community Setting
- Risk-Based Approach for Treatment of Myelofibrosis
- Addressing the Challenges of Peripheral T-Cell Lymphoma Management
- Cold Agglutinin Disease (CAD): The Role of the Complement Pathway in Treating a Rare Autoimmune Hemolytic Anemia
- The Era of Disease-Modifying Therapy for Duchenne Muscular Dystrophy: Outlining Care Pathways for the Evolving Needs of the Patient
- Pediatric Epilepsy HCP Session 3
- Pediatric Epilepsy HCP Session 2
- Hemophilia Caregiver - Full program
- Pediatric Epilepsy Caregiver Q&A
- Pediatric Epilepsy Caregiver Module 6
- Pediatric Epilepsy Caregiver Module 5
- Pediatric Epilepsy Caregiver Module 4
- Pediatric Epilepsy Caregiver Module 3
- Pediatric Epilepsy Caregiver Module 2
- Pediatric Epilepsy Caregiver Module 1
- SMA Caregiver Module 6
- SMA Caregiver Module 5
- SMA Caregiver Module 4
- SMA Caregiver Module 3
- SMA Caregiver Module 2
- SMA Caregiver Module 1
- SMA HCP Program
- Hemophilia A 6/18 HCP
- Pediatric Epilepsy
- Hairy Cell HCP
- SMA HCP
Event Agendas
- Summit Virtual 2020
- Living Rare, Living Stronger 2020 - Virtual Agenda v2
- Living Rare, Living Stronger 2020 - Virtual Agenda
- Living Rare, Living Stronger 2020
COVID-19 Resources
- COVID-19 Vaccine Information
- mRNA Vaccines for COVID-19
- Keep Receiving Treatment During COVID-19: Online Addiction Recovery Resources
- NORD & FDA Listening Session: COVID19 Impact on Rare Disease Communities
- The Impact of the COVID-19 Pandemic on the Rare Disease Community
- Social Media, Misinformation and the COVID-19 Pandemic
- Building a Village: How to Re-Engage Your Board Members, Leaders and Volunteers
- COVID-19-Associated Multisystem Inflammatory Syndrome in Children
- Share Your COVID-19 Telehealth Experience
- Fundraising in a Pandemic
- The Impact of COVID-19 on Clinical Trials
- Activating Your Medical Advisors for COVID19 Response - Slide Deck
- Coalition letter asking the Centers for Medicare and Medicaid services to provide flexibility to Medicare Advantage and Prescription Drug Plan sponsors to waive the use of step therapy requirements during the COVID-19 pandemic
- Crisis Communications: COVID-19 and the Future
- Coalition letter urging Congressional leadership to strengthen the Medicaid program in future legislation to address the COVID-19 pandemic
- Coalition letter to the White House urging an end to all surprise medical billing
- Coalition letter to Congressional Leadership urging an end to all surprise medical billing
- COVID-19 Rapid Response Leadership Series
- Coalition letter urging HHS to issue additional guidance to prevent discrimination for triaging and health care during COVID-19
- CMS COVID-19 Stakeholder Engagement Calls Archive
- Rare Policy Response to COVID-19
- NORD COVID-19 Community Survey Infographic
- NORD COVID-19 Community Survey Report
- Coalition letter to Louisiana Governor Edwards requesting the state adopt a policy that refrains from discriminating against people with disabilities and chronic conditions during the COVID-19 emergency
- Coalition letter to Connecticut Governor Lamont on the state’s document titled “Standards of Care: Providing Health Care During A Prolonged Public Health Emergency and requesting that he continue to work with the disability and chronic care community as the state develops additional guidance during COVID-19
- Engaging Your Board and Leaders in Response to Crisis
- Financial Strategies during COVID-19
- Crash Course in Remote Management PDF
- Coalition letter to New Hampshire Governor Sununu requesting the state adopt a policy that refrains from discriminating against people with disabilities and chronic conditions during the COVID-19 emergency
- Coalition letter to Senate leadership urging the inclusion of bridge funding for stalled research in the next COVID-19 relief package
- Coalition letter to HHS urging allowance of copay assistance to count toward annual cost sharing in 2021 Notice of Benefit & Payment Parameters
- Telehealth Information Guide
- A list of 60 digital resources on mental health, from Social Work License Map.
- Coalition letter to New York Governor Cuomo to express concerns over the state’s ventilator allocation guidelines during COVID-19
- Coalition letter to New Jersey Governor Murphy highlighting concerns in recent guidance and requesting that he continue to work with the disability and chronic care community as the state develops additional guidance during COVID-19
- Coalition letter to Texas Governor Abbott requesting the state adopt a policy that refrains from discriminating against people with disabilities and chronic conditions during the COVID-19 emergency
- COVID-19: Current Status of Testing, Treatments and Vaccines
- Strategies for Coping During the COVID-19 Pandemic
- Coalition letter to Florida Governor Desantis requesting the state adopt a policy that refrains from discriminating against people with disabilities and chronic conditions during the COVID-19 emergency
- Coalition letter to Virginia Governor Northam requesting the state adopt a policy that refrains from discriminating against people with disabilities and chronic conditions during the COVID-19 emergency
- NORD Organizational Database
- NORD Rare Disease Reports
- Coalition statement of support for H.R. 6514, the Worker Health Coverage Protection Act
- Rare Action Network (RAN) COVID-19 Virtual Discussion Groups
- NIH COVID-19 Social Media Resources
- Called to Action: NORD's Response to COVID-19
- Coalition to Congressional leadership urging guardrails on the use of step therapy protocols in Medicare and Medicaid during COVID-19
- Updated guidance on grace periods for binder and premium payments
- Information on special enrollment periods
- COBRA coverage continuation Q&A
- COBRA coverage and the Marketplace
- Medicaid and CHIP coverage
- Losing job based coverage
- Health coverage options if you are unemployed
- Telemedicine Letters to Governors
- Strategies for Coping with Loneliness and Isolation During the Coronavirus Pandemic
- Social Security Administration website
- Microsoft COVID-19 Resource Guide for Nonprofits
- Coalition letter Urging HHS to Prevent Discrimination For Triaging and Health Care During COVID-19
- Come hang out with The Mighty
- Talking with Children about Coronavirus Disease 2019
- Meditation techniques from Headspace.com
- National Alliance on Mental Illness COVID-19 Resource and Information Guide
- National Institutes of Health: COVID-19 Updates
- Online courses from the World Health Organization
- Rolling updates from the World Health Organization on COVID-19
- People with serious illness can teach us about living with COVID-19
- Evolving medical coverage and drug administration updates for those needing clinical care.
- Understanding the Landscape of COVID-19 Vaccine and Treatment R&D
- Coalition letter to Congress to further strengthen health insurance coverage, affordability, and accessibility in the next COVID-19 relief package
- COVID-19 Policy Issues Contact Us Form
- Mindful Parenting
- Meditation Apps for Kids
- Coronavirus: Mental Health Coping Strategies
- Talking with Children about Coronavirus Disease 2019
- 12 Simple Ways to Teach Mindfulness to Kids
- What Nonprofit Board Members Should Be Doing Right Now to Address the COVID-19 Situation
- How Nonprofits and Board Members Can Respond to COVID-19
- Nonprofits and Coronavirus
- COVID-19: Resources for Nonprofit Leaders
- FDA Coronavirus Disease Resources for Patients
- CDC Coronavirus Page
- Social Distancing Struggles?
- COVID-19 (Coronavirus Disease 19)
- Coronavirus Preparedness: What Do Caregivers Need to Know?
- Coronavirus Preparedness for People with Chronic Diseases
- Mental Health And COVID-19 – Information And Resources
- Happy - 24/7
- Center for Chronic Illness
- Event Emergency Preparedness Guide
- Activating Your Medical Advisors for COVID19 Response
- Fundraising: Building your Case with Foundations and Industry
- NORD/EURORDIS-Rare Diseases Europe joint statement on COVID-19 and orphan drug legislation
- Letter to state governors, medicaid directors and insurance commissioners
- Letter to Congress on home infusion access for medicare beneficiaries
- Letter to Congress asking for flexibility for patients filling prescriptions during COVID-19
- Families First Coronavirus Response Act
- Coalition statement urging the Administration to create a special enrollment period for the healthcare.gov marketplace
- Coalition letter to HHS and CMS urging decisive action to slow the spread of COVID-19 among people with pre-existing conditions and vulnerable populations
- Coalition letter in support of including the Protecting Seniors Through Immunization Act in the next COVID-19 response legislative package
- Join the Rare Action Network
- COVID-19 State by State Resources
- A Rare Response: Addressing the COVID-19 Pandemic
- NORD Asks A Rare Disease Expert: COVID-19
Speakers
- Bert Bruce
- Kristen Angell
- Lydia Shenouda
- Stephen Hahn
- Oodaye Shukla
- Francois Nader
- Martin Mackay
- Maha Katabi
- Jason Hafler
- David Scheer
- Dennis V. Chornenky
- Nick Bath
- Stuart Portman
- Khair El Zarrad
- Kira Mann
- Linda Gouler Blount
- James Gellar
- Kay Holcombe
- Christine Munro
- Fritz Bittenbender
- Randy Milby
- Robin Kelley
- Charles Holmes
- Suneet Varma
- Lisa Anne Thompson Taylor
- Lisa Sarfaty
- Alice Houk
- Paul Solari
- Debra Regier
- Julian Isla
- Mike Porath
- Mark Sleeper
- Corrie Painter
- Peter Langmuir
- Ashley Ward
- Jane Larkindale
- Phillip Tai
- Martin Ho
- Larissa Lapteva
- Janet Maynard
- Hylton Joffe
- Hannah Armstead
- Kevin Mott
- Kirk Campbell
- Eric Dube
- David Fajgenbaum
- Jim Palma
- Vishal Bhatnagar
- John Hopper
- Peter Marks
- Charles Venditti
- Karlyne Reilly
- Walter Koroshetz
- Christopher Austin
- Brooke Thomas
- Willam Marsh
- William Brady
- Susan Berry
- Yann le Cam
- Tanisha Carino
- Durhane Wong-Rieger
- Deanna Portero
- Theresa Strong
- Sandra Sermone
- Miriam O'Day
- Melissa Bryce Gamble
- Debbie Drell
- Vanessa Vogel-Farley
- Heidi Ross
- Julie Breneiser
- Camilla Lauritzen
- Shoshana Shendelman
- Terence Sanger
- Kate Zhang
- Laura Trutoiu
- Kristi Mitchell
- Mark Yale
- Telba Irony
- Vanessa Boulanger
- Danyelle Sun
- Karmen Trzupek
- Susan Manganaro
- David Keane
- Danielle Boyce
- Pamela Gavin
- Rachel Sher
- Monica Webb Hooper
- Kathleen Sullivan
- Michelle McMurry-Heath
- Adam Gluck
- Shafali Spurling Jeste
- Jennifer Beck
- Amy Medina
- Peter Saltonstall
Program Resources
- RareLaunch Toolkit (December 2020 Workshop)
- Create a Rare Disease Advisory Council in Your State (Video)
- Creating an RDAC in Your State Toolkit
- RDAC One Pager
- RDAC Stakeholders Meeting Recording
- Research Ready Quick Reference Guide
- Get Inspired to Take the Leap to Form a 501(c)(3) or Establish a Disease Registry to Advance Your Community
- Fundraising
- IAMRARE Registry Program
- Activating Your Medical Advisors for COVID-19 Response
- Building Your Case: Fundraising for Your Organization
- Fundraising: Activate Your Board
- Increase Donations with Financial Transparency
- Board Management
- Natural History Studies and Patient Registries
- COVID-19 Rapid Response Leadership Series
- Tip Sheet for Medical Advisory Board
- Policy and Procedures for Working with a Medical Advisory Board Template
- Medical Advisory Board Member Agreement Template
- Privacy Policy Template
- Policy on Actual and Potential Conflicts of Interest Template
- Board Electoral Process and Term Limits Language Template
- Bylaws General Nonprofit Structure Template
- Operating Budget Template
- Website Financial Disclosure Guide
Event Posters
- PaVe-GT: Paving the Way for Rare Disease Gene Therapies
- Seeing to the Continuity of Healthcare in an era of Physical Distancing
- Sarcoma Coalition We Are Here For You
- Payer Survey Highlights Confusion About the Definition and Utility of Real World Evidence in Rare Disease
- Managing Online Survey as a Successful Global Rare Disease Patient Community Building Model
- GM2DR – The Tay Sachs and Sandhoff Disease Multinational Patient Reported Data Registry
- Building a Global Rare Disease Coalition Beneficial Outcomes From the Establishment of an International Alliance Community
- An Update on Treatment Data from the Desmoid Tumor Research Foundation
- Understanding Parent and Expert Concerns and Experiences in Rett Syndrome Qualitative Research to Inform Outcome Measure Development
- Resilience Education Supporting the Emotional Wellbeing of Those with a Progressive Disease
- Pharmacist-identified Gaps in the Expanded Access Process at an Academic Medical Center
- Accelerating Research Through A Patient-led Collaborative Network
- Update in the Management of Bell's Palsy
- Academic and Community Collaboration to Accelerate Kabuki Syndrome Awareness, Research, and Clinical Care
- Patient Advocate-Driven Advancement of Clinical Recommendations in Ultra-Rare Disease The CLN1 Batten Disease Experience
- Granulomatosis with Polyangiitis A Rare Condition with Otolaryngologic Manifestations
- Educational Impact of Digital Learning Spinal Muscular Atrophy Education for Clinicians and Caregivers
- Characteristics of Generalized Pustular Psoriasis (GPP) Patients Results from the Optum® Clinformatics™ Data Mart Database
- Access for Patients and Caregivers
- A Model of Medical Education That Teaches Doctors to #DareToThinkRare
- Fostamatinib, an Oral Spleen Tyrosine Kinase (SYK) Inhibitor, as Second-Line Therapy for Immune Thrombocytopenia (ITP)
- RT001 in Progressive Supranuclear Palsy (PSP) In-Vitro and Clinical Results in 3 Patients
- Physical Function Clinical Outcome Assessments for Rare Disorders and Sarcopenia Planning Core Outcome Sets with FDA
- Next-Generation Phenotyping (NPG) for Cat-Eye Syndrome
- Integrated Study Results of Teprotumumab The First Drug US FDA Approved for the Treatment of Thyroid Eye Disease
- Identifying Causative Variants in Patients with Monogenic Stone Disease
- Fostamatinib for the Treatment of Warm Antibody Autoimmune Hemolytic Anemia (wAIHA) Results of the Phase 2, Open-Label Study
- Effects of Bacterial Products on PMP Tumor Cells
Organizations
- Coalition to Cure Calpain 3
- Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders
- Neuroacanthocytosis Advocacy USA, Inc.
- The Bonnell Foundation: Living with Cystic Fibrosis
- Xia-Gibbs Society
- Transplant Unwrapped
- Wolfram Syndrome UK
- DeSanto-Shinawi Syndrome Corp
- National CMV Foundation
- CACNA1A Foundation
- KIF4A Foundation
- Canadian Association of Pompe
- Malan Syndrome Foundation
- Avery’s Hope
- Timothy Syndrome Alliance
- STXBP1 Foundation
- Kindness for Kimberlee
- Achalasia Awareness Organization
- Potentials Foundation, INC
- The Myelin Project
- Neuropathy Trust
- Neuropathy Association
- Aicardi Syndrome Awareness and Support Group
- Endosalpingiosis Foundation Inc
- Noah’s Hope–Hope4Bridget Foundation
- Duplication Cares
- Cure Sanfilippo Foundation
- The CGD Association of America
- Child and Youth Care
- Tatton Brown Rahman Syndrome Community
- Smith-Kingsmore Syndrome Foundation
- The EHE Foundation
- Project 8p
- ALD Connect
- Yellow Brick Road Project
- Sam Day Foundation
- Plan for Rare Foundation
- The Snow Foundation
- Global DARE Foundation
- Neuromuscular Disease Foundation
- Sickle Cell Association of Texas Marc Thomas Foundation
- Foundation to Fight H-abc
- Rare Disease Ghana Initiative
- The Cute Syndrome Foundation
- Superficial Siderosis Research Alliance, Inc.
- Pulmonary Alveolar Proteinosis Foundation, Inc.
- Our Odyssey
- Pompe Support Network
- Gaucher Community Alliance
- Conquering Gyrate Atrophy
- GACI Global
- International Autoimmune Encephalitis Society
- Syngap Research Fund
- NEC Society
- Arrivederci ALD
- LGMD-1D DNAJB6 Foundation and International Registry
- Myhre Syndrome Foundation
- Noonan Syndrome Foundation
- Chris CJ Johnson Foundation Inc.
- Kidney Cancer Research Alliance
- Melanoma Research Foundation
- ASXL Rare Research Endowment (ARRE) Foundation
- Multiple Sulfatase Deficiency Action Foundation
- GRIN2B Foundation
- Helping Hands for GAND, Inc.
- NORD's™ Rare Cancer Coalition (RCC)
- Cure AP-4
- Sickle Cell Association of Houston, Inc.
- Sara’s Cure
- Tyrosinemia Society, Inc.
- Remember The Girls
- KCNB1.org
- FOXG1 Research Foundation
- Cure VCP Disease, Inc.
- National Median Arcuate Ligament Syndrome Foundation, Inc.
- Avery's Angels Gastroschisis Foundation
- Liv4TheCure
- Dreamsickle Kids Foundation
- Perthes Kids Foundation
- CMT Research Foundation (CMTRF)
- AXYS
- Hepatitis B Foundation
- Chronic Recurrent Multifocal Osteomyelitis Foundation (CRMO)
- IRF2BPL Foundation
- Ovarian Cancer Research Alliance
- Transient Global Amnesia Project
- Klippel-Feil Syndrome Freedom
- Keep Swimming Foundation
- Salla Treatment And Research
- SLC6A1 Connect
- Mackenzie's Mission
- Turner Syndrome Global Alliance
- Hypersomnia Foundation
- FamilieSCN2A Foundation
- CHAMP1 Research Foundation
- The Aneurysm and AVM Foundation
- Organization for Rare Diseases India
- DHPS Foundation
- CSNK2A1 Foundation
- Global Hydranencephaly Foundation
- The Avalon Foundation
- International Neuroendocrine Cancer Alliance
- No Stomach For Cancer
- Dysautonomia Support Network
- SETBP1 Society
- International Sacral Agenesis/Caudal Regression Association (iSACRA)
- COPA Syndrome Foundation
- RUNX1 Research Program
- The Sumaira Foundation for NMO
- Arizona Network for PKU and Allied Disorders
- Pompe Warrior Foundation
- Sick Cells
- iSEEK Pulmonary Hypertension Hope Center
- Connecting Families Urea Cycle Disorders Foundation
- Thisbe and Noah Scott Foundation
- Gould Syndrome Foundation (COL4a1/COL4A2)
- Bili Project Foundation
- ADCY5.org
- NORSE Institute
- Cauda Equina Foundation, Inc.
- KrabbeConnect
- KAT6A Foundation
- Legg Calve Perthes Foundation
- MCT8-AHDS Foundation
- Intractable Childhood Epilepsy Alliance
- Pheo Para Alliance
- Danny’s Dose Alliance
- TANGO2 Research Foundation
- The Visual Snow Initiative
- The Brain Recovery Project: Childhood Epilepsy Surgery Foundation
- FND Hope
- Open Medicine Foundation
- NBIAcure
- Rare Kids Network
- Defeat MSA Alliance
- Neurofibromatosis Northeast
- Team Audrey
- A Cure for Ellie
- AlphaNet, Inc.
- KIF1A.ORG
- Illness Challenge Foundation
- Amniotic Fluid Embolism Foundation (AFE Foundation)
- Advocacy & Awareness for Immune Disorders Association (AAIDA)
- Lung Transplant Foundation
- HCU Network America
- Atypical HUS Foundation
- Yash Gandhi Foundation Finding a Cure for I-Cell
- Canadian Hepatopulmonary Syndrome Program
- United MSD Foundation
- Hajdu Cheney Syndrome Support Group
- Foundation for USP-7 Related Diseases
- Mila’s Miracle Foundation, Inc.
- The Aarskog Foundation
- XP Family Support Group
- The Jansen’s Foundation
- People Against Leigh Syndrome
- Monarch Initiative
- International FOXG1 Foundation
- Little People UK
- All Things Kabuki
- In-Depth Diagnostics
- ADNP-Kids Research Foundation
- Asociacion de Sindrome Miastenico Congenito - Congenital Myasthenic Syndrome Association
- Gastroparesis Patient Association for Cures and Treatments, Inc.
- PHACE Syndrome Community
- Li-Fraumeni Syndrome Association
- Talia Duff Foundation, Inc.
- Hyper IgM Foundation, Inc.
- MotherToBaby
- Recurrent Meningitis Association
- Genetic Disorders UK
- Rare Cancer Genome Consortium
- BORN A HERO, Pfeiffer’s Health and Social Issues Awareness
- PMD Family Support
- MLD Foundation
- PURA Syndrome Foundation
- Blepharospasm Australia Inc.
- Cheyanna's Champions 4 Children
- HSAN1E Society
- Annabelle's Challenge
- Sofia Sees Hope
- Clusterbusters, Inc.
- The Calliope Joy Foundation
- National Leiomyosarcoma Foundation
- The DDX3X Foundation Fund
- Xtraordinary Joy
- Adrenal Insufficiency United (AIU)
- Amyloidosis Research Consortium, Inc.
- FPIES Foundation
- Larsen Syndrome Resource Center
- Parent to Parent New Zealand, Inc.
- Consortium of Multiple Sclerosis Centers
- Canadian PBC Society
- Sanfilippo Children’s Foundation
- Evans Syndrome Foundation
- Imerman Angels One-on-One Cancer Support
- PCD Foundation
- Canadian CMTC Foundation
- RYR-1 Foundation
- Chloe’s Fight Rare Disease Foundation
- Sitosterolemia Foundation
- Siegel Rare Neuroimmune Association
- Autoimmune Encephalitis Alliance
- Spinal CSF Leak Foundation
- National Health Council
- Charlotte & Gwenyth Gray Foundation to Cure Batten Disease
- Mitochondrial Medicine Society
- International Society for Cutaneous Lymphomas
- KCNQ2 CURE Alliance
- Bohring-Opitz Syndrome Foundation, Inc.
- NICER Foundation
- Rare & Undiagnosed Network
- OMSLife Foundation
- Curing Retinal Blindness Foundation
- Autoimmune Hepatitis Association
- Portuguese Association for CDG
- Pachyonychia Congenita Project
- Mowat-Wilson Syndrome Foundation
- Fibrolamellar Cancer Foundation (FCP)
- MTM-CNM Family Connection, Inc.
- ZNM- Zusammen Stark! e. V.
- A Twist of Fate-ATS
- Sweet's Syndrome UK
- Bartter Syndrome Foundation
- HCU Network Australia
- A Cure in Sight
- SBS Cure Project
- Child Growth Foundation
- Rett Syndrome Research Trust
- The JMML Foundation
- CMTC Alliance
- Red Sanfilippo AC
- Tess Research Foundation
- Snyder-Robinson Foundation
- Martin Mueller IV Achalasia Awareness Foundation, Inc.
- Bridge the Gap – SYNGAP Education and Research Foundation
- TKO Strong Foundation, Inc.
- Rothmund-Thomson Syndrome Foundation
- National ALS Registry
- Rare Cancer Research Foundation
- Indian Organization for Rare Diseases
- Hereditary Leiomyomatosis & Renal Cell Cancer Family Alliance
- Daybreak Children's Rare Disease Fund
- Worldwide Syringomyelia & Chiari Task Force, Inc.
- TargetCancer Foundation
- Lighthouse International
- Pyruvate Kinase Deficiency Group
- Kids Wish Network
- PRP Alliance, Inc.
- Pitt Hopkins Research Foundation
- Hereditary Colon Cancer Foundation
- Fat Disorders Resource Society
- Ocular Melanoma Foundation
- Jared's Juggernaut, Inc.
- Julia’s Wings Foundation, Inc.
- Community United for Research and Education of Ocular Melanoma
- BCM Families Foundation
- Bardet Biedl Syndrome Family Association
- Oligophrenin-1 Syndrome Foundation
- NTM Info & Research, Inc.
- PCDH19 Alliance
- Gut Check Foundation
- RASopathies Network USA
- Team Telomere, Inc.
- International FH Foundation
- Relapsing Polychondritis Awareness and Support Foundation Inc.
- Bloom's Syndrome Association, Inc.
- Beautiful You MRKH Foundation, Inc.
- We Are R.A.R.E.
- Focus On Rhabdo
- Rare Trait Hope Fund
- MPN Education Foundation
- Pediatric Arachnoid Cyst Foundation
- Lymphatic Malformation Institute
- Pseudohypoaldosteronism Foundation
- UKD Foundation
- Health Agency of Canada
- Genetic and Rare Disorders Organisation
- International Foundation for Autoimmune Arthritis
- Brittle Diabetes Foundation Inc.
- WonderBaby.org
- Association for Creatine Deficiencies
- Epidermoid Brain Tumor Society
- Cortical Foundation
- PMG Awareness Organization, Inc.
- Little Miss Hannah Foundation
- cureCADASIL
- SSADH Association (Succinic Semialdehyde Dehydrogenase Deficiency)
- Campbell Burns Metabolic Trust
- Ring14 USA Outreach, Inc.
- Vision of Children Foundation
- Infogen AC
- Myotubular Trust
- Potocki-Lupski Syndrome Outreach Foundation, Inc
- Hereditary Leiomyomatosis and Renal Cell Cancer (HLRCC) Family Alliance
- International Hip Dysplasia Institute
- The Courtney Project, Inc.
- Genetic Disease Foundation
- PVNH Support & Awareness
- PANDAS Network.org
- Alex TLC (The Leukodystrophy Charity)
- Global FKRP Registry
- LGMD2I Research Fund
- National Ophthalmic Disease Genotyping and Phenotyping Network (eyeGENE®)
- International Alliance of Patients' Organizations
- Wilhelm Foundation
- Lysosomal Storage Disorders Support Society
- Center for Information and Study on Clinical Research Participation
- Parent to Parent USA
- Circadian Sleep Disorders Network
- Arthrogryposis Multiplex Congenita Support, Inc.
- Joshua Frase Foundation
- Cancer Legal Resource Center
- Myelin Disorders Bioregistry Project (MDBP)
- Usher Syndrome Coalition
- National Stem Cell Foundation
- Williams Syndrome Family of Hope
- International FPIES Association
- PTEN World
- Danielle's Hope, Inc.
- FORCE: Facing Our Risk of Cancer Empowered
- Sarcoidosis & Lyme Disease Support Australia
- National Sarcoidosis Organization
- Answering T.T.P. (Thrombotic Thrombocytopenic Purpura) Foundation
- MEBO Research, Inc.
- Glut1 Deficiency Foundation
- Breathtakers OB Trust
- BHD Foundation
- LAL Solace, Inc.
- Huntington's Disease Youth Organization
- PHA EUROPE, European Pulmonary Hypertension Association
- Skull Base Institute
- Loey Dietz Syndrome Foundation
- Liam's Land Organization, Inc
- ROHHAD Fight Inc.
- Stiff Man Syndrome Support group
- Rare Genomics Institute
- RE Children's Project, LLC
- Rare Diseases Clinical Research Network
- Hong Kong Mucopolysaccharidoses & Rare Genetic Diseases Mutual Aid Group
- Program for the Study of Neurodevelopment in Rare Disorders
- Hope for Hypothalamic Hamartomas
- Proyecto Pide un Deseo México, i.a.p.
- Dysautonomia Youth Network of America, Inc.
- European Network for Ichthyosis (ENI)
- Neuroscience Research Australia
- Bladder Exstrophy Research Foundation
- AKU Society of North America
- Cleft Lip and Palate Foundation of Smiles
- Foundation for Research on Rare Diseases and Disorders
- Foundation for Peripheral Neuropathy
- Foundation for Angelman Syndrome Therapeutics (FAST)
- Short Bowel Syndrome Foundation, Inc.
- Global Healthy Living Foundation (GHLF)
- Oral Cancer Foundation
- M-CM Network
- Cure JM Foundation
- Chordoma Foundation
- American Multiple Endocrine Neoplasia Support
- Global Foundation for Peroxisomal Disorders
- Lymphedema Clinic Wittlinger Therapy Center
- PHG Foundation
- Retroperitoneal Fibrosis World Support
- Nathaniel Adamczyk Foundation
- 1p36 Deletion Support & Awareness
- Soft Bones, Inc.: The US Hypophosphatasia Foundation
- NET Patient Foundation
- Focus Foundation, Inc.
- Ramon Brugada Senior Foundation
- Myrovlytis Trust
- Rare Kidney Stone Consortium
- Taylor Bug Kisses Foundation
- Jonahs Just Begun - Foundation to Cure Sanfilippo, Inc.
- International 22q11.2 Deletion Syndrome Foundation, Inc.
- Dupuytren Research Group
- International Palindromic Rheumatism Society
- Benign Essential Blepharospasm Canadian Research Foundation, Inc.
- F.I.R.M.O. Fondazione Raffaella Becagli
- Childhood Liver Disease Research and Education Network
- Cystinosis Foundation UK
- United States Bone and Joint Decade
- Simons Searchlight
- Centre for Genetics Education
- Medical Home Portal
- Acromegaly Community, Inc.
- Thalassemia Support Foundation
- Association for Children with a Disability
- United Survivors with Epidermolysis Bullosa
- Coeliac Society of Australia Inc.
- Cure2Children Foundation
- International Association for Medical Assistance to Travellers
- Movement Disorder Society
- The FD/MAS Alliance
- Lynch Syndrome International
- Living Beyond Breast Cancer
- Chemical Injury Information Network
- Jack McGovern Coats Disease Foundation
- International Foundation for CDKL5 Research
- MitoAction
- CID - Central Institute for the Deaf
- Patient AirLift Services (PALS)
- Who is Amy?
- Australasian Blistering Diseases Foundation
- DanonDisease.org
- Dravet Syndrome Foundation, Inc.
- Sudden Unexplained Death In Childhood (SUDC) Foundation
- Stiff Person Syndrome.Net
- Living With Stiff Person Syndrome
- CLOVES Syndrome Community
- Beyond Batten Disease Foundation
- Northwest Sarcoma Foundation
- G6PD Deficiency Association [Associazione Italiana Favismo, onlus]
- Huntington's Disease Society of America - Connecticut Affiliate
- International Brain Tumour Alliance
- Bachmann-Strauss Dystonia & Parkinson Foundation
- GA GALT
- Association for Multiple Endocrine Neoplasia Disorders (AMEND)
- Variety - The Children's Charity of the United States
- Marc Lustgarten Pancreatic Cancer Foundation (dba The Lustgarten Foundation)
- Bear Necessities Pediatric Cancer Foundation
- Norwegian Porphyria Centre
- Pleuropulmonary Blastoma Research at Children's National Medical Center
- TransActive Education & Advocacy
- Cochrane Collaboration
- CURE: Citizens United for Research in Epilepsy
- Wisconsin Chiari Center
- Cholangiocarcinoma Foundation
- Hearing Loss Association of America
- Northwest Parkinson's Foundation
- International League Against Epilepsy
- Sanfilippo Foundation Switzerland
- CJ Foundation for SIDS, Inc.
- International Hyperhidrosis Society
- National Neuromuscular Centre, Norway
- Sudden Infant Death Services of Illinois, Inc.
- North American Hair Research Society
- United Cerebral Palsy
- Global Fibrosis Foundation
- Rare Disease UK
- NCL Resource
- Group of Alliance, Investigation and Support in Rare Diseases
- Guthy-Jackson Charitable Foundation
- National PKU Alliance
- Congenital Hyperinsulinism International
- Urea Cycle Disorders Consortium
- International Prader Willi Syndrome Organization
- Child Neurology Foundation
- Training, Education & Research Institute, Inc.
- MPN Research Foundation
- National Health Information Center
- Smile Foundation
- Share Pregnancy & Infant Loss Support, Inc.
- Hospitality Homes
- Cook for Love, Inc.
- Fetal Hope Foundation
- Familial Dysautonomia Now Foundation
- National Fabry Disease Foundation
- Camp Twin Lakes
- SeriousFun Children's Network
- Bobby Jones Chiari & Syringomyelia Foundation
- Foundation Building Strength for Nemaline Myopathy
- Alport Syndrome Foundation
- National Brain Tumor Society
- WIRED4LIFE
- Hemispherectomy Foundation
- National Patient Travel Center
- Fanconi Hope
- Myositis Support Group at the Hospital for Special Surgery
- European Skeletal Dysplasia Network
- Matthew's Friends
- LGS Foundation, Inc.
- Muscular Dystrophy Association of Kosovo
- PLAN Institute for Caring Citizenship
- International Mesenteric Panniculitis Society
- Myotonic Dystrophy Foundation
- Through the Looking Glass
- Appendix Cancer/Pseudomyxoma Peritonei Research Foundation (ACPMP)
- Cystinosis Research Foundation
- CureDuchenne
- MSS Research Foundation (Marshall-Smith Syndrome)
- Conquer Chiari
- Accord Alliance
- Cogan's Contact Network
- Mastokids
- Corneal Dystrophy Foundation
- Cure CMD (Congenital Muscular Dystrophy)
- Kidney & Urology Foundation of America, Inc.
- International Stills Disease Foundation, Inc.
- Ectodermal Dysplasia Society
- LAM Health Project
- ECD Global Alliance
- International Society for Systemic Auto-Inflammatory Diseases
- Townes-Brocks Syndrome International Support Network
- Patient Advocate Foundation Co-Pay Relief
- Fundaci�n FOP
- BioMed Central Ltd
- Spinal Muscular Atrophy Foundation
- Syndromes Without A Name USA
- CORE
- M.O.R.G.A.N. Project
- BeatSarcoma
- Infants Remembered In Silence, Inc. (IRIS)
- Hannah's Hope Fund
- Sickle Cell Awareness Group of Ontario
- National Center on Deaf-Blindness
- Childhood Eye Cancer Trust
- Perkins School for the Blind
- DuchenneConnect
- United States Adult Cystic Fibrosis Association, Inc.
- Hemihypertrophy Support
- Cornea Research Foundation of America
- International Hydranencephaly Support Group
- Livestrong Foundation
- Vitiligo Support International
- Janine Sarcoidosis Outreach Foundation
- STARS
- Young Women's Breast Cancer Program
- Desmoid Tumor Research Foundation
- Beyond Celiac
- Lung Cancer Alliance
- Breath of Hope, Inc.
- Dandy-Walker Alliance, Inc.
- Myocarditis Foundation
- Lymphangiomatosis & Gorham’s Disease Alliance, Inc. (LGDA)
- Cancer Support Community
- Cancer.Net
- Matrix Parent Network and Resource Center
- Tarlov Cyst Disease Foundation
- American Association of Neurological Surgeons
- International Cystinuria Foundation
- Adenoid Cystic Carcinoma Research Foundation
- UCSF Fibrodysplasia Ossificans Progressiva Clinic
- UCSF Hemophilia Treatment Center
- UCSF Pulmonary Hypertension Clinic
- Retina International
- Hide & Seek Foundation for Lysosomal Disease Research
- Autoinflammatory Alliance
- United Pompe Foundation
- Multiple Sclerosis Foundation
- Adult Medical Genetics Program, University of Colorado
- Boston Foundation for Sight
- Hughes Syndrome Foundation
- Supporting Aussie Kids with Kabuki Syndrome
- Global and Regional Asperger's Syndrome Partnership (GRASP)
- Children with Spinal Muscular Atrophy, Ukraine - Kharkiv Charitable Foundation
- Friends of Cancer Research
- Cold Agglutinin Disease Foundation
- American Partnership for Eosinophilic Disorders (APFED)
- Jain Foundation Inc.
- APBD Research Foundation
- APS Type 1 Foundation
- European Society for Immunodeficiencies
- PSC Partners Seeking a Cure
- Endometriosis Research Center
- Living Resources
- Philippine Society for Orphan Disorders
- Claire Altman Heine Foundation, Inc.
- Vermont Assembly of Home Health Agencies
- American Academy of Audiology
- Children's Interstitial Lung Disease Foundation
- Bartter Site
- KickAS.org
- PLGA Foundation
- Fight SMA/Spinal Muscular Atrophy
- International Psychogeriatric Association
- Cure Alveolar Soft Part Sarcoma International
- Association for Frontotemporal Degeneration (AFTD)
- Patient Registries at Slone: Myeloma & MDS
- Trichorhinophalangeal Syndrome Association
- Cincinnati Center for Eosinophilic Disorders
- Speech-Language and Audiology Canada
- Campaign Urging Research for Eosinophilic Disease (CURED)
- Taking Control of Your Diabetes
- XLP Research Trust
- Advancement of Research for Myopathies (ARM)
- Neuroleptic Malignant Syndrome Information Service, Inc.
- Nationwide Children's Hospital
- Advocacy for Neuroacanthocytosis Patients
- Pituitary Foundation
- Fight ALD
- Amyloidosis Foundation
- Foundation for Prader-Willi Research
- Amyloidosis Australia
- Amyloidosis Support Groups, Inc.
- Pediatric Brain Tumor Foundation
- Aicardi Syndrome Foundation
- Fair Foundation
- Hidradenitis Suppurativa Foundation, Inc
- Sneddon Foundation (Stichting Sneddon)
- Neuroendocrine Tumor Research Foundation
- Huntington's Association of South Africa
- Mowat Wilson Support Group
- International Scleroderma Network
- PMD Foundation
- National Blood Clot Alliance
- ThyCa: Thyroid Cancer Survivors' Association, Inc.
- Spasmodic Torticollis ST/Dystonia, Inc.
- UCSF Memory and Aging Center
- DEBRA International
- Kleine-Levin Syndrome Foundation
- Myotonic Dystrophy Support Group
- Self-Help Information Service of Nebraska
- Beckwith-Wiedemann Children's Foundation International
- iFace
- Irish Chronic Pain Association
- APS Foundation of America
- National Parkinson Foundation, Inc.
- Fragile X Society
- Irish Haemophilia Society
- FSH-Muscular Dystrophy Support Group
- Spina Bifida Hydrocephalus Queensland
- Let Them Hear Foundation
- SADS UK (Sudden Arrhythmic Death Syndrome)
- Mouth Cancer Foundation
- Irish Heart Foundation
- Addison's Disease Self-Help Group (UK)
- Congenital Adrenal Hyperplasia Support Group
- CAH Support Group
- Pancreatitis Supporters Network
- Macular Disease Society
- Gorlin Syndrome Group
- Child Brain Injury Trust
- Spinal Cord Tumor Association, Inc.
- National Deaf Children's Society
- National Meningitis Association
- Locks of Love
- Alkaptonuria Society
- Ellis-van Creveld Syndrome Web Site
- Melorheostosis Association
- Spina Bifida & Hydrocephalus Association of Ontario
- Center for Peripheral Neuropathy
- Kharkov Renaissance Foundation For Children With Spinal Muscular Atrophy
- P.A.R.C. Promoting Awareness of RSD/CRPS in Canada
- Phelan-McDermid Syndrome Foundation
- Churg Strauss Syndrome Association
- New England Hemophilia Association
- National Creutzfeldt-Jakob Disease Research & Surveillance Unit
- GIST Support International
- Esophageal Cancer Awareness Association, Inc.
- Atresia/Microtia Online E-mail Support Group
- Foundation Fighting Blindness (Canada)
- Sickle Cell Disease Foundation of California
- Guillain-Barre Syndrome Foundation of Canada, Inc.
- Geneva Centre for Autism
- National Organization for Disorders of the Corpus Callosum
- The Multiple System Atrophy Coalition, Inc.
- Histiocytosis Association of Canada
- Manitoba Ankylosing Spondylitis Association
- Mid-Atlantic Connection for PKU and Allied Disorders, Inc.
- Lupus Society of Alberta
- Henshaws Society for Blind People
- ME Research UK
- Instituto de Errores Innatos del Metabolismo
- Angel Flight Southeast, Inc.
- Genetic and Rare Diseases (GARD) Information Center
- American Association of the Deaf-Blind
- International Bureau for Epilepsy
- Pulmonary Fibrosis Foundation
- GIST Cancer Research Fund
- New Horizons Un-Limited, Inc.
- Christopher & Dana Reeve Foundation
- Support Organisation for Trisomy and Related Disorders
- Congenital Adrenal Hyperplasia Education and Support Network
- Inflammatory Breast Cancer Research Foundation
- International Painful Bladder Foundation
- American Vitiligo Research Foundation, Inc.
- Gorlin Syndrome Alliance
- MdDS Balance Disorder Foundation
- Fetal Alcohol Spectrum Disorders Center for Excellence
- XXYY Project
- Bowel Group for Kids Inc.
- Turner Syndrome Support Society (UK)
- Erythema Nodosum Yahoo Support Group
- PNH Support Group
- PCD/KS Foundation
- Alpha-1 Research Registry
- MD Support - The Eyes of the Macular Degeneration Community
- EDS Today
- NephCure Kidney International Foundation
- Wisconsin ME/CFS Association, Inc.
- Infant Botulism Treatment and Prevention Program
- OT Resource
- Periodic Paralysis International
- Cicatricial Alopecia Research Foundation (CARF)
- Lewy Body Dementia Association, Inc.
- ITP Support Association
- Preeclampsia Foundation
- National Alliance for Hispanic Health
- ALS Therapy Development Institute
- Carol Ann Foundation
- DDC Clinic for Special Needs Children
- Behavior Education Training Associates
- Dubowitz Syndrome Support
- Degos Patients' Support Network
- Dystonia Europe
- European Multiple System Atrophy Study Group
- Pelizaeus Merzbacher Disease Support Group
- International Oral Lichen Planus Support Group
- Fibromuscular Dysplasia Society of America
- Aniridia Network UK
- Rare Diseases Denmark
- Pathways Awareness Foundation
- Leukodystrophy Australia
- Pediatric Brain Tumors
- National Prion Disease Pathology Surveillance Center
- National Eosinophilia Myalgia Syndrome Network
- Stichting ERFO-centrum
- World Arnold Chiari Malformation Association
- Bone Marrow Foundation
- International Paruresis Association
- R.O.C.K. (Raising Our Celiac Kids)
- Swedish Association of Rare Disorders
- Federacion Espanola de Enfermedades Raras
- Propionic Acidemia Foundation
- Belgian Association for Metabolic Diseases
- Genetic Alliance UK
- Encephalitis Society
- HS-USA, Inc.
- CJD Aware!
- ITP Foundation
- Texas Neurofibromatosis Foundation
- About Madelungs
- Genetics Center, Inc.
- International Alliance of ALS/MND Associations
- Distal Trisomy 10q Families
- Cutaneous Lymphoma Foundation
- Children's Fund for Glycogen Storage Disease Research, Inc.
- Hemophilia Federation of America
- Canavan Foundation
- Save Babies Through Screening Foundation
- International Pleuropulmonary Blastoma Registry
- Lymphatic Education & Research Network
- Health Information Network
- Multiple Myeloma Research Foundation
- Research!America
- Rothberg Institute For Childhood Diseases
- Hereditary Neuropathy Foundation, Inc.
- Crigler-Najjar Association/King's Way Foundation
- Genetic Support Network of Victoria
- Oto Palatal Digital Syndrome Family Resource Network
- Sotos Syndrome Support Association of Canada
- Life Raft Group
- Hormone Health Network Endocrine Society
- Cystic Fibrosis Worldwide
- Eye Cancer Foundation
- Rare Cancer Alliance
- Genetic and Rare Conditions Site
- Genetics Education Center
- Juvenile Diabetes Research Foundation International
- Mathew Forbes Romer Foundation, Inc.
- CJD Voice
- A. J. Pappanikou Center for Excellence in Developmental Disabilities
- Association for Retinopathy of Prematurity and Related Diseases
- BC Neurofibromatosis Foundation
- International Mosaic Down Syndrome Association
- Cystinosis Research Network, Inc.
- Foundation for Thymic Cancer Research
- Associazione Internazionale Ring 14
- Cardio-Facio-Cutaneous International (CFC)
- Angioma Alliance
- Spastic Paraplegia Foundation
- Cochrane Cystic Fibrosis and Genetic Disorders Group
- Tom Wahlig Foundation
- Children's Cardiomyopathy Foundation
- Canavan Research Illinois
- Hairy Cell Leukemia Foundation
- Sarcoma Alliance
- National Shingles Foundation
- aHUS Alliance
- Helen Keller National Center for Deaf-Blind Youths and Adults
- Sarcoma Foundation of America
- Adenoid Cystic Carcinoma Organization International
- CHARGE Family Support Group
- Organization for Understanding Cluster Headaches (O.U.C.H.)
- American Nystagmus Network, Inc.
- Foundation for Sarcoidosis Research
- Xeroderma Pigmentosum Support Group
- Garrod Association of Canada
- Tetrasomy/Pentasomy X Support Group
- International Advocate For Glycoprotein Storage Diseases
- Kennedy’s Disease Association, Inc.
- Mesothelioma Applied Research Foundation
- British Porphyria Association
- Niemann-Pick Disease Group (UK)
- Hypermobility Syndrome Association
- National Pesticide Information Center
- EB Medical Research Foundation
- New Zealand Organization for Rare Disorders
- Lysosomal Diseases New Zealand
- Aphasia Hope Foundation
- Congenital Heart Information Network (C.H.I.N.)
- Newlife Foundation for Disabled Children
- Pancreatica.org---Confronting Pancreatic Cancer
- Taste and Smell Health Center at University of Connecticut
- Center for Hope
- Birth Defect Research for Children, Inc.
- Sturge Weber Foundation (UK)
- Leukaemia CARE
- Canavan Research Foundation
- SWAN UK
- Children's Neurobiological Solutions Foundation
- CGD Society
- Headlines - Craniofacial Support Group
- Taiwan Foundation for Rare Disorders
- Cutis Laxa Internationale
- American Society for Experimental NeuroTherapeutics
- CARES Foundation, Inc.
- CMTC-OVM Cutis Marmorata Telangiectatica
- Daisy Network
- Intracranial Hypertension Research Foundation
- Vascular Cures
- Stichting Erfocentrum
- Familial Dysautonomia Hope Foundation
- MND Scotland
- Mitochondria Research Society
- Cancer Research UK
- Children's Glaucoma Foundation
- San Diego Brachial Plexus Network
- Choroideremia Research Foundation, Inc.
- Apraxia Kids
- VISIONS Services for the Blind and Visually Impaired
- Foundation for Women's Cancer
- Prader-Willi France
- Parents of Infants and Children with Kernicterus (PICK)
- Marinesco-Sjogren Syndrome Support Group
- Ehlers-Danlos Support Group UK
- Encephalitis Global
- Pancreatic Cancer Action Network
- Foundation For A Cure For Mitochondrial Disease, Inc.
- National CFIDS Foundation, Inc.
- Ocular Immunology and Uveitis Foundation
- Shaken Baby Alliance
- Organisation For Anti-Convulsant Syndrome
- National Batten Disease Registry
- HITS (UK) Family Support Network
- Asherman's Syndrome Online Community
- Children's Gaucher Research Fund
- Pediatric Keratoplasty Association
- Worster-Drought Syndrome Support Group
- National Academy of Elder Law Attorneys, Inc.
- Nail Patella Syndrome Worldwide
- Eye Cancer Network
- International Foundation for Optic Nerve Disease (IFOND)
- Dattoli Cancer Foundation
- Hunter's Hope Foundation, Inc.
- Barth Syndrome Foundation
- NIH/Clinical Trials
- CADASIL-Together We Have Hope
- Platelet Disorder Support Association
- Canadian Lymphedema Framework
- International WAGR Syndrome Association
- Pediatric Neurotransmitter Disease Association
- Iron Disorders Institute
- Wheelchairnet
- STARS - Syncope Trust & Reflex Anoxic Seizures
- American Association for Klinefelter Syndrome Information and Support
- Little Hearts, Inc.
- Kids With Heart ~ National Association for Children's Heart Disorders, Inc.
- Engle Laboratory at Children's Hospital Boston
- Kidneeds
- Angel Flight NE
- United Brachial Plexus Network
- US Hereditary Angioedema Association, Inc.
- Society for Mucopolysaccharidoses and Related Disorders
- Floating Harbor Syndrome Support Group
- XLH Network Inc.
- Infectious Diseases Society of America
- The Balanced Mind Foundation
- Solve ME/CFS Initiative
- Alpha-1 Foundation
- Adult Congenital Heart Association
- goldenhar Syndrome Support Network Society
- Partners Resource Network
- American Hemochromatosis Society
- MHE and Me- A Support Group for Kids with Multiple Hereditary Exostoses
- Associazione Italiana Mucopolisaccaridosi E Malattie Affini
- National Women's Health Information Center
- Friedreich’s Ataxia Research Alliance
- Lesch-Nyhan Disease International Study Group
- Human Genome Project Information Web Site
- International Society of Nurses in Genetics (ISONG)
- American College of Medical Genetics
- American Society of Human Genetics
- Information for Genetic Professionals
- Genetic Alliance Australia
- Carter Centers for Brain Research in Holoprosencephaly and Related Malformations
- NIH/National Institute of Child Health and Human Development
- Children's Heart Society
- Cancer Care, Inc.
- Frontotemporal Dementia Support Group
- National Society for Phenylketonuria (UK)
- Vulvar Pain Foundation
- Gilda Radner Familial Ovarian Cancer Registry
- National Emphysema Foundation
- British Liver Trust
- Healing Exchange Brain Trust
- International Cancer Alliance for Research and Education (ICARE)
- Mended Hearts, Inc.
- Worldwide Cancer Research
- Swedish Hemophilia Society
- Muscular Dystrophy Ireland
- Multiple Sclerosis Society of Ireland
- European Alliance of Neuromuscular Disorders Associations
- Multiple Sclerosis Society UK
- Multiple Sclerosis Society of Canada
- North American Menopause Society
- Multiple Sclerosis International Federation
- International Ostomy Association
- Blind Citizens Australia
- Arthritis Foundation of Australia
- Heart Foundation of Australia
- Eating Disorders Association of Queensland
- Childhood Cancer Association, Inc.
- Lymphoedema Association of Australia
- Irritable Bowel Information & Support Association of Australia, Inc.
- Juvenile Diabetes Foundation Australia
- Muscular Dystrophy Australia
- Lung Foundation Australia
- SIDS and Kids Australia
- Spinal Cord Injury Alberta
- Ovacome (UK)
- Myaware (UK)
- Meningitis Research Foundation (UK)
- United Ostomy Association of Canada
- National Association of Laryngectomee Clubs (UK)
- Diabetes New Zealand
- Mental Health Foundation (UK)
- Prostate Cancer UK
- Lymphoma Action (UK)
- Canadian Hearing Society
- Ovarian Cancer National Alliance
- M.A.G.N.U.M. Migraine Awareness Group: A National Understanding for Migraineurs
- Lymphoma Canada
- Hepatitis C Society of Canada
- Canadian Celiac Association
- International Diabetes Federation
- World Federation for Mental Health
- SANE Australia
- Schizophrenia Society of Canada
- Diabetes UK
- Lung Association
- Canadian Cancer Society
- Canadian Diabetes Association
- Heart and Stroke Foundation of Canada
- Disability Rights Nebraska
- Fibromyalgia Association UK
- International Society for Minimally Invasive Cardiac Surgery
- Society of Interventional Radiology
- Academy of Nutrition and Dietetics
- CureSearch for Children's Cancer
- Ileostomy and Internal Pouch Support Group
- Lupus UK
- Epilepsy Foundation of Victoria
- British Lung Foundation
- RP Fighting Blindness
- Nystagmus Network (UK)
- British Acoustic Neuroma Association
- National Association for Colitis & Crohn's Disease (UK)
- Lymphoedema Support Group - Tasmania
- Council for Responsible Genetics
- Catastrophic Illness in Children Relief Fund Commission
- Pathfinder Parent Center
- Disability Rights Network of Pennsylvania
- Kentucky Protection and Advocacy
- Ontario Association for Families of Children with Communication Disorders
- Anaphylaxis Campaign
- FOLKS: Friends of Landau Kleffner Syndrome
- Lymphoma Research Foundation
- American Society for Gastrointestinal Endoscopy
- American Orthopaedic Society for Sports Medicine
- American Medical Women's Association
- American Congress of Rehabilitation Medicine
- American Academy of Ophthalmology - The Eye M.D. Association
- National Board For Respiratory Care, Inc.
- American Hospital Association
- Society for Investigative Dermatology
- American Thoracic Society
- Society of Nuclear Medicine
- American College of Emergency Physicians
- Society of Critical Care Anesthesiologists
- American Society for Histocompatibility & Immunogenetics
- American Society of Tropical Medicine and Hygiene
- American Society for Colposcopy and Cervical Pathology
- American Trauma Society
- Society for the Study of Reproduction
- American Society of Andrology
- Carcinoid Cancer Foundation
- American Society For Dermatologic Surgery
- Disability Rights Idaho
- American Association for Geriatric Psychiatry
- American Academy of Pediatric Dentistry
- American Pediatric Association
- American Society of Transplantation
- American Association for Cancer Research
- American Society of Nephrology
- American Society of Colon and Rectal Surgeons
- American Society of Anesthesiologists
- American Psychoanalytic Association
- American Psychiatric Association
- American Otological Society
- American Academy of Family Physicians
- American Academy of Neurology
- American Academy of Orthopaedic Surgeons
- American Academy of Pediatrics
- American Academy of Physical Medicine And Rehabilitation
- American Association of Clinical Endocrinologists
- American Association of Immunologists
- American Gastroenterological Association
- Society of Gynecologic Oncologists
- American Association of Gynecologic Laparoscopists
- American College of Gastroenterology
- American Neurological Association
- National Association of Psychiatric Health Systems
- American College of Surgeons
- American College of Chest Physicians
- American Board of Psychiatry and Neurology, Inc.
- American Board of Ophthalomology
- American Congress of Obstetricians and Gynecologists
- American College of Rheumatology
- American Academy of Facial Plastic And Reconstructive Surgery
- American Academy of Child & Adolescent Psychiatry
- American Board of Medical Specialties
- Israeli Huntington Disease Association
- Equip For Equality, Inc.
- Advocacy Center (Louisiana)
- Disability Law Center (Utah)
- Ellis-van Creveld Syndrome Foundation
- Fundacion Distonia (Dystonia)
- New Zealand Dystonia Patient Network Inc.
- Vermont Legal Aid
- Mental Health America of Illinois
- NVACP (Dutch Federation of Addison and Cushing Patients and CAH and Conn)
- Disability Rights North Carolina
- Montana Advocacy Program
- European Organization for Research and Treatment of Cancer
- New Jersey Self-Help Clearinghouse
- Alström Syndrome International
- Sleep/Wake Disorders Association of Toronto
- Osteoporosis 2000
- Microcephaly Support Group
- Cystic Fibrosis Research, Inc.
- Lymphovenous Association of Ontario
- PMP Pals' Network
- Triple X Support Group
- CMT United Kingdom
- Sjögren's Syndrome Association
- International Glaucoma Association
- National Cervical Cancer Coalition
- Alström Syndrome UK
- Moebius Syndrome Association
- Project Assist (Nevada)
- Kabuki Syndrome Network
- Huntington's Disease Association of New Zealand
- Canadian Hemochromatosis Society
- United Self-Help (Hawaii)
- Nevada PEP
- Alveolar Capillary Dysplasia Association
- Ataxia-Telangiectasia Society (UK)
- Fundaci�n Alfa-1 de Puerto Rico
- Project DOCC - Delivery of Chronic Care
- Foundation for Blood Research
- Reach
- Crohn's and Colitis Foundation of Canada
- Children's Mitochondrial Disease Network
- SHARE - Self-Help For Women With Breast or Ovarian Cancer
- Canadian Reflex Sympathetic Dystrophy Network
- CONVERSATIONS! The International Ovarian Cancer Connection
- National Ovarian Cancer Coalition
- Miami Project to Cure Paralysis
- International Huntington Association
- Thrombophilia Awareness Project
- euro-ATAXIA
- Progressive Supranuclear Palsy Association UK
- Hepatitis Foundation International
- Children's Brittle Bone Foundation
- Erythromelalgia Association
- Chromosome 22 Central
- Canadian Association for Community Living
- British Sjögren’s Syndrome Association
- Association for Glycogen Storage Disease (UK) Ltd
- NBIA Disorders Association
- Purine Metabolic Patients' Association
- Central Brain Tumor Registry of the United States
- Vascular Birthmarks Foundation
- Disorders of Chromosome 16 Foundation
- goldenhar Family Support Group
- Endocrine Society
- Cleft Lip and Palate Association
- Canadian Immunodeficiencies Patient Organization
- Canadian Addison Society
- ARPKD/CHF Alliance
- American Skin Association
- Canadian Liver Foundation
- Primary Biliary Cholangitis Organization
- National Ankylosing Spondylitis Society
- Health Connect of South Dakota
- Meningitis Foundation of America
- Trichotillomania Learning Center
- Romberg's Connection
- OBGYN.net: The Obstetrics & Gynecology Network
- Pityriasis Rubra Pilaris Support Group
- Sarcoidosis Online Sites (S.O.S.)
- Micro & Anophthalmic Children's Society
- Selective Mutism Group
- NIH/National Heart, Lung and Blood Institute ~ Hematology Branch
- Nevus Outreach, Inc.
- Myocarditis Program at Mayo Clinic
- Council for Exceptional Children
- Hereditary Tyrosinemia Group (Groupe Aide Aux Enfants Tyrosinemiques Du Quebec)
- North American Riding for the Handicapped Association
- Center For Loss In Multiple Birth, Inc.
- Canine Companions for Independence
- Hemangioma Support System
- Paralyzed Veterans of America
- Association of Community Cancer Centers
- Center for Research on Women with Disabilities
- American Association for Continuity of Care
- Starlight Children's Foundation
- Parents Anonymous, Inc.
- American Association of Tissue Banks
- National Organization on Disability
- National Perinatal Association
- American Institute of Stress
- Fight for Sight, Inc.
- Toxic Discovery
- Sail To Prevail
- Trisomy 12p Parent Support Organization
- Gerontological Society of America
- National Council on Patient Information and Education
- KIDSCOPE
- Schiz Kidz Buddies - Schizencephaly Support Group
- National Institute for Rehabilitation Engineering
- Periodic Paralysis Association
- Think First Foundation
- Children's Wish Foundation International, Inc.
- Pacific Southwest Regional Genetics Network
- Hydrocephalus Support Group, Inc.
- Living Bank International
- Epilepsy Canada
- Postpartum Support International
- National Shared Housing Resource Center
- Obsessive Compulsive Anonymous
- Children's Cancer & Blood Foundation
- Disability Rights Education and Defense Fund
- Children's Leukemia Research Association
- Homeless Resource Center
- United States Society for Augmentative and Alternative Communication
- International Ventilator Users Network
- ALS Society of Canada
- American Epilepsy Society
- Baby's Breath
- Dysautonomia Foundation, Inc., Toronto Chapter
- Parents of Vision Impaired (NZ), Inc.
- Perspectives Network, Inc.
- Parentpals.com
- National Safety Council/Environmental Health Center
- International Association for the Study of Pain
- BrightFocus Foundation
- National Association of State Mental Health Program Directors
- Juvenile Diabetes Research Foundation
- Ara Parseghian Medical Research Foundation
- Tourette Syndrome Foundation of Canada
- Ontario Federation for Cerebral Palsy
- Robinow Syndrome Foundation
- Lupus Canada
- Nemaline Myopathy Support Group
- Rehabilitation Research & Training Center on Aging with Developmental Disabilities
- Matheny School and Hospital
- Canadian Fanconi Anemia Research Fund
- National Organization of Social Security Claimants' Representatives
- MOVE International
- Washington State Fathers Network
- American RSDHope
- Aplastic Anemia & Myelodysplasia Association of Canada
- United Spinal Association
- Canadian Angelman Syndrome Society
- GeneTests
- Sisters Network, Inc.
- Acoustic Neuroma Association of Canada
- Nail-Patella Syndrome (UK)
- GADA CANADA
- Tuberous Sclerosis Canada Sclerose Tubereuse
- Center for Narcolepsy, Sleep and Health Research
- AED Pregnancy Registry - Massachusetts General Hospital
- Proteus Syndrome Foundation
- Cincinnati Center for Development Disorders
- European Chromosome 11q Network
- Canadian Osteogenesis Imperfecta Society
- Center For Disfigurement
- Comer Children's Hospital - University of Chicago (Neurofibromatosis Clinic)
- National Institute for Occupational Safety and Health
- Wegner Health Sciences Information Center
- PAVE
- Selective Mutism Foundation, Inc.
- PATH Parent to Parent of CT
- Family Support Network of North Carolina
- International Institute of Reconstructive Microsurgery
- Vereniging voor Kinder met Stofwisselingsziekten
- Human Brain and Spinal Fluid Resource Center
- Well Spouse Association
- Munroe-Meyer Institute for Genetics and Rehabilitation
- ORPHANET
- Pain.com
- Shriners Hospitals for Children
- NIH/Consensus Development Program
- Juvenile Dermatomyositis Registry at Children's Memorial Hospital
- Pediatric/Adolescent Gastroesophageal Reflux Association, Inc.
- NIH/Office of Research on Women's Health
- Jeffrey Modell Foundation
- Dana Alliance for Brain Initiatives
- Second Wind Lung Transplant Association, Inc.
- Creutzfeldt-Jakob Disease Foundation, Inc.
- Stuttering Foundation of America
- Raynaud's Association, Inc.
- Scottish Society for Autism
- American Medical Association DoctorFinder
- Hospice Foundation of America
- AboutFace International
- National Eczema Society UK
- Kidney Cancer Association
- Autism Research Institute
- Genetic Alliance
- Multiple Sclerosis Association of America
- Prostate Pointers
- Medicare Rights Center
- EURORDIS- Rare Diseases Europe
- International Pemphigus & Pemphigoid Foundation
- Stevens Johnson Syndrome Foundation
- NDI (Nephrogenic Diabetes Insipidus) Foundation
- National Keratoconus Foundation
- National Association for Adults with Special Learning Needs
- NKH International Family Network
- MHE Coalition
- Malignant Hyperthermia Investigation Unit
- Spinal Muscular Atrophy Support UK
- Hypertrophic Cardiomyopathy Association
- Erb's Palsy Group
- Cardiomyopathy Association
- Brain Tumor Foundation for Children, Inc.
- Blind Children's Center
- Association Francophone ds Glycogenoses
- Anorchidism Support Group
- American SIDS Institute
- "22q and You" Center
- Achromatopsia Network
- International Patient Organization for Primary Immunodeficiencies
- OncoLink: The University of Pennsylvania Cancer Center Resource
- TransWeb
- FOD (Fatty Oxidation Disorders) Family Support Group
- Helping Hands: Monkey Helpers for the Disabled
- Healing the Children Northeast, Inc.
- Guide Dogs of America
- Gauchers Association (UK)
- Family Voices, Inc.
- Disabled Sports USA
- Pet Partners
- Danish Centre for Rare Diseases and Disabilities
- Dandy-Walker Syndrome Network
- Compassionate Friends - Supporting Family After A Child Dies
- Compassion & Choices
- Children's Organ Transplant Association, Inc.
- Children's Brain Tumor Foundation
- Montgomery Heart Foundation for Cardiomyopathy
- Hope After Loss, Inc.
- 11q Research and Resource Group
- Muscular Dystrophy Association of New Zealand, Inc.
- Support for People with Oral and Head and Neck Cancer, Inc.
- Vanderbilt's Autonomic Dysfunction Center
- Guillain-Barre & Associated Inflammatory Neuropathies
- International Registry of Werner Syndrome
- DebRA
- Disabled Peoples' International
- The Brain Tumour Charity
- Childhood Cancer Canada Foundation
- Brain Tumour Foundation of Canada
- Shine
- International Costello Syndrome Support Group
- Angel View Crippled Children's Foundation, Inc.
- AIS-DSD Support Group
- Food Allergy Research & Education
- Health Resource Center for Women with Disabilities
- Association Fransaise Contre l'amylose
- Les Turner Amyotrophic Lateral Sclerosis Foundation, Ltd.
- Cancer Hope Network
- Miracle-Ear Children's Foundation
- MSUD Family Support
- National Bone Marrow Transplant Link
- Ronald McDonald House Charities
- CDG Care
- Horses of Hope Oregon
- Federation for Children with Special Needs
- Disabled American Veterans
- Center for Persons with Disabilities
- Michigan Diabetes Research and Training Center
- Craniofacial Foundation of America
- Carroll Center for the Blind
- The Arc of Greater Cleveland
- Children's Liver Association for Support Services (C.L.A.S.S.)
- Magazine for Families with Members Having Special Needs
- CenterWatch, Inc.
- Birth to Three Developmental Center
- American Council on Rural Special Education
- Special Care Dentistry
- American Geriatrics Society
- American Art Therapy Association, Inc.
- Acid Maltase Deficiency Association (AMDA)
- American Bar Association Commission on Mental and Physical Disability Law
- Mercy Medical Angels
- ALS Association-Greater Philadelphia Chapter
- West Central Missouri Community Action Agency
- Progressive Osseous Heteroplasia Association
- National Library of Medicine
- Fabry Support & Information Group
- National Captioning Institute
- Described and Captioned Media Program
- United States Access Board
- Hyperacusis Network
- UNIQUE - Rare Chromosome Disorder Support Group
- Dystonia Society
- National Autistic Society
- Foundation for Spinal Cord Injury Prevention, Care and Cure
- Tracheo Oesophageal Fistula Support
- National Sjogren's Syndrome
- Tuberous Sclerosis Association
- Turner Syndrome Society of Canada
- Albinism Fellowship
- EA/TEF Child and Family Support Connection, Inc.
- NIH/National Asthma Education and Prevention Program
- Memorial Sloan-Kettering Cancer Center
- American Academy of Allergy, Asthma and Immunology
- Social Security Administration
- Office of Disability Employment Policy
- National Council on Disability
- President's Committee for People with Intellectual Disabilities
- Office of Special Education and Rehabilitative Services
- Office of Fair Housing/Equal Opportunity
- National Library Service for the Blind and Physically Handicapped
- Indian Health Service
- United States Department of Health and Human Services
- Substance Abuse and Mental Health Services Administration
- Health Resource and Services Administration
- RTC Portland - Family Support and Children's Mental Health & Training Center
- NIH/National Institute on Deafness and Other Communication Disorders
- Office of Minority Health
- National Center for Health Statistics
- Agency for Healthcare Research and Quality
- Neuroblastoma Children's Cancer Society
- National Coalition for Cancer Survivorship
- National Breast Cancer Coalition
- Erythropoietic Protoporphyria Research and Education Fund
- M. D. Anderson Cancer Center Hereditary Colorectal Cancer Registry
- Northwestern University Feinberg School of Medicine
- Ferguson Hospital
- Delaware Cancer Registry
- Southeastern Hereditary Colorectal Cancer Registry
- Amtrak Special Needs & Accessibility
- Greyhound Customers with Disabilities Travel Assistance
- Dancing Eye Syndrome Support Trust
- South Nashville Family Resource Center
- Rasmussen's Syndrome and Hemispherectomy Support Network
- The LAM Foundation
- Neutropenia Support Association, Inc.
- National Pediatric Neuroinflammation Organization, Inc.
- Learning Disabilities Association of Canada
- Human Ecology Action League, Inc. (HEAL)
- Immunization Action Coalition
- Oley Foundation
- Pull-Thru Network
- Gastro-Intestinal Research Foundation
- NIH/National Institute of General Medical Sciences
- Family Resource Centers Network of California
- NF-2 Sharing Network
- MedicAlert Foundation International
- NIH/Osteoporosis and Related Bone Diseases National Resource Center
- New England Consortium of Metabolic Programs
- Massachusetts General Hospital Neurofibromatosis Clinic
- University of Toronto
- Massachusetts General Department of Anesthesiology
- Jefferson University Medical School
- University of Texas Medical Branch at Galveston
- Mayo Clinic Dept. of Anesthesiology
- National Veterans Services Fund
- American Organ Transplant Association
- Zero to Three
- Children's Hospice International
- Center for Assistive Technology
- Children's Liver Disease Foundation
- Danish Apert Syndrome Association (Danmarks Apertforening)
- Disabled Children's Relief Fund
- Dogs for the Deaf, Inc.
- Lupus Research Alliance
- Cri Du Chat Syndrome Support Group
- Crisis Clinic (Helping Lives On The Line)
- American Celiac Society - Dietary Support Coalition
- Intensive Caring Unlimited, Inc.
- American Council for Headache Education
- Disabled and Alone/Life Services for the Handicapped, Inc.
- National Association for the Dually Diagnosed
- International Waldenstrom’s Macroglobulinemia Foundation
- Orphan Europe
- European Commission
- American Sexual Health Association
- Together for Short Lives
- Atlantis Community, Inc.
- The Association for the Severely Handicapped
- MENCAP
- RADAR - The Disability Network
- Psoriasis and Psoriatic Arthritis Alliance
- American Urological Association, Inc.
- Kennedy Institute of Ethics
- Center for International Blood and Marrow Transplant Research
- Institute for Human Services/Helpline
- House Ear Institute
- National Institute of Environmental Health Sciences
- Food and Drug Administration (FDA) Office of Orphan Products Development
- Patient Advocates for Advanced Cancer Treatments
- American Occupational Therapy Association, Inc.
- National Center for Environmental Health Strategies, Inc.
- International Foundation for Functional Gastrointestinal Disorders
- National Rehabilitation Association
- New Directions for People with Disabilities, Inc.
- National Perinatal Information Center
- John Tracy Clinic for Preschool Deaf Children
- Job Accommodation Network
- Iowa Division of Persons with Disabilities
- Touch Trust
- International Hearing Dog, Inc.
- Galactosaemia Support Group
- Michigan Protection and Advocacy Service, Inc.
- Mainstream, Inc.
- CHERUBS - The Association of Congenital Diaphragmatic Hernia Research, Awareness and Support
- National Association of Protection and Advocacy Systems
- Telecommunications for the Deaf and Hard of Hearing, Inc.
- ASK About * Special * Kids
- Endometriosis Association
- Craniofacial Clinic at University of Virginia
- American Spinal Injury Association
- Xeroderma Pigmentosum Society, Inc.
- World Health Organization (WHO)
- World Federation of Hemophilia
- US TOO International, Inc.
- University of Southern California Medical Center
- Washington University School of Medicine
- University of Pennsylvania Dermatology Clinic
- Thyroid Foundation of Canada
- Spina Bifida and Hydrocephalus Canada
- MyFace
- Society for Muscular Dystrophy Information International
- Canadian Society for Mucopolysaccharide and Related Diseases, Inc.
- Society for Mucopolysaccharide Diseases
- Smith-Magenis Syndrome Foundation
- Sickle Cell Association of Ontario
- Rush University Medical Center
- Rubinstein-Taybi Syndrome, UK Support Group
- RESOLVE: National Infertility Association
- Learning Ally
- Scleroderma and Raynaud's UK
- Rheumatology Outpatient Clinic at Beth Israel Hospital
- Galactosemia Foundation
- Parent Education Network
- NIH/Recombinant DNA Program
- NIH/Office of Rare Disease Research
- National Cancer Institute
- NIH/National Institute of Dental and Craniofacial Research
- NIH/National Kidney and Urologic Diseases Information Clearinghouse
- NIH/National Institute of Neurological Disorders and Stroke
- NIH/National Institute of Mental Health
- NIH/National Institute on Drug Abuse
- NIH/National Institute of Diabetes, Digestive & Kidney Diseases
- NIH/National Institute of Allergy and Infectious Diseases
- NIH/Institute on Alcohol Abuse and Alcoholism
- NIH/National Institute on Aging
- NIH/National Heart, Lung and Blood Institute
- NIH/National Eye Institute
- NIH/National Institutes of Health
- NIH/National Human Genome Research Institute
- National Cancer Institute Physician Data Query
- NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases
- NIH/Child Health and Human Development - Section on Endocrinology and Genetics
- Georgetown University Medical Center
- Children's National Medical Center
- Children's Hospital of Philadelphia Neurofibromatosis Program
- Cedars-Sinai Medical Genetics Institute
- Neurofibromatosis Clinic at Texas Children's Hospital
- Klinefelter Syndrome Association of Canada
- Alternating Hemiplegia of Childhood Foundation (AHCF)
- International Federation for Spina Bifida and Hydrocephalus
- International Fanconi Anemia Registry
- Institute of Reconstructive Plastic Surgery (New York Medical University)
- Williams Syndrome Foundation
- Huntington Society of Canada
- George Washington University HEATH Resource Center
- Guiding Eyes for the Blind, Inc.
- Online Mendelian Inheritance in Man
- Fragile X Association of Michigan
- Glaucoma Research Foundation
- Fertility Research Foundation
- Eye Bank Association of America
- Arthritis Society
- National Eczema Association
- Tufts Medical Center
- Neurological Institute of New York
- Digestive Disease National Coalition
- DBA-UK
- Diamond Blackfan Anemia Foundation
- Association For Bladder Exstrophy Community
- National Stuttering Association
- National Spinal Cord Injury Association
- Myositis Support Group
- Prevent Blindness America
- Cystic Hygroma and Lymphangioma Support Group
- Cystic Fibrosis Trust
- Department of Human Genetics/Cri du Chat
- Craniofacial Pain Center
- Mountain States Genetics Foundation
- National Hospice and Palliative Care Organization
- National Hansen's Disease Program
- National Foundation of Dentistry For the Handicapped
- National Eye Care Project
- National Disease Research Interchange
- BackCare, the Charity for Healthier Backs
- National Association of the Deaf
- Socialserve.com
- Clearinghouse on Disability Information
- Chronic Granulomatous Disease Registry
- Centers for Disease Control and Prevention
- SAMHSA's Center for Substance Abuse Prevention
- Canadian Organization for Rare Disorders
- Canadian Hemophilia Society
- Canadian Cystic Fibrosis Foundation
- Be the Match Registry
- British Polio Fellowship
- Organic Acidaemias UK
- Rose Kushner 's Women's Breast Cancer Advisory Center
- Harvard Brain Tissue Resource Center
- Brain Injury Association of America
- NICHD Brain and Tissue Bank for Developmental Disorders
- Restricted Growth Association
- Center for Housing and New Community Economics (CHANCE)
- European Leukodystrophy Association
- Angelman Syndrome Support Education and Research Trust
- ME Association
- Courage Center
- Muscular Dystrophy Campaign
- Muscular Dystrophy Canada
- American Society of Plastic Surgeons
- Muscular Dystrophy Association
- American Society of Parenteral and Enteral Nutrition
- American Society of Hypertension
- American Society of Dermatopathology
- Motor Neurone Disease Association
- American Rhinologic Society
- The Mastocytosis Society, Inc.
- American Lung Association
- American Printing House for the Blind
- American Hearing Research Foundation
- Academy of Spinal Cord Injury Professionals, Inc.
- American Medical Association
- Make-A-Wish Foundation of America
- American Diabetes Association
- American Dental Association
- Lifetime Advocacy Plus
- American Cancer Society, Inc.
- American Association of Orthodontists
- American Academy of Dermatology
- Boggy Creek Gang
- International Center for Disability Resources on the Internet
- Educational Equity Center at The Academy for Educational Development
- Caregiver Action Network
- Sexuality Information and Education Council of the U.S.
- International Hearing Society
- National Foundation For Transplants
- Chronic Illness Coalition
- Volunteer Center, Inc.
- Hastings Center
- Nebraska Family Support Network
- SupportWorks
- Selfhelp
- Westchester Self-Help-Clearinghouse
- Recovery International
- Northwest Regional Self-Help Clearinghouse
- Worldwide Disability Solutions Group
- National Stroke Association
- Cruse Bereavement Care
- National Arts & Disability Center
- Prader-Willi Syndrome Association (UK)
- Deaf and Disabled Telecommunications Program
- Paradigm Healthcare Services
- Life'sWork
- San Diego Regional Center for the Developmental Disabilities
- Riverwood Center
- Pilot Parents of Southern Arizona
- Center for Assistive Technology and Environmental Access
- Sundial Special Vacations, Inc.
- Simon Foundation for Continence
- United Way Information & Referral Services
- National Association for the Education of Young Children
- Sibling Support Project
- Contact A Family
- Support Organization for Trisomy 13/18 and Related Disorders, UK
- Indoor Air Quality Information Clearinghouse
- American Association of Suicidology
- Family Support Center
- Transplant Recipients International Organization, Inc.
- Blanche Fischer Foundation
- Stickler Involved People
- Cushing’s Support and Research Foundation, Inc.
- Iowa Regional Genetic Consultation Service
- National Transplant Assistance Fund
- Stickler Syndrome Support Group
- Severe Chronic Neutropenia International Registry
- Southeastern Regional Genetics Group, Inc.
- National Kidney Foundation Serving Connecticut
- Li-Fraumeni Syndrome International Registry
- National Healthy Mothers, Healthy Babies Coalition
- SIDS Resources
- Seattle Children's Hospital
- Department of Human Genetics at Mt. Sinai Medical Center
- Opsoclonus-Myoclonus Support Network, Inc.
- Family Caregiver Alliance
- Sudden Arrhythmia Death Syndromes Foundation
- Siblings for Significant Change
- Cystinosis Foundation, Inc.
- National Alliance on Mental Illness
- Pacific Northwest Regional Genetics Group
- The Arc of the Capital Area
- National Aphasia Association
- The Arthrogryposis Group
- Cure HHT Foundation
- Metabolic Clinic at The Children's Hospital
- Wolf Hirschhorn Syndrome Trust for the UK and Ireland
- International Myeloma Foundation
- Neurogenetic Surgery Center at Massachusetts General Hospital
- DHMC Genetics and Child Development Center
- Clinical Genetics Program, Children's Hospital Boston
- PHP - Parents Helping Parents, Inc.
- International Dyslexia Association
- Skin Cancer Foundation
- Easter Seals
- Division of Genetics at Georgetown University Medical Center
- American Heart Association
- Sotos Syndrome Support Association
- Division of Medical Genetics, Department of Medicine, UC San Diego
- Parent Project Muscular Dystrophy
- American Sickle Cell Anemia Association
- Intestinal Multiple Polyposis and Colorectal Cancer Registry
- Cobalamin Network
- Johns Hopkins Hereditary Colorectal Cancer Registry
- FOCUS Families
- Feingold Center for Children
- Organic Acidemia Association
- PKD Foundation
- New York State Office for People with Developmental Disabilities
- International Center for Fabry Disease
- CurePSP, Inc.
- Progeria Research Foundation, Inc.
- National Rosacea Society
- American Council of the Blind
- Crohn's and Colitis Foundation of America
- Pituitary Network Association
- International Lesch-Nyhan Disease Association
- Gluten Intolerance Group of North America
- Epilepsy Foundation
- Spinal Cord Society
- National Neutropenia Network
- Better Hearing Institute
- Center for Sickle Cell Disease
- Post-Polio Health International
- American Foundation for the Blind
- American Sleep Apnea Association
- Arthritis Foundation
- Chromosome 18 Registry & Research Society
- LymeDisease.org
- National Women's Health Network
- Yale Child Study Center
- Aarskog Syndrome Parents Support Group
- American Lung Association in Connecticut, East Hartford
- American Society for Deaf Children
- Dup 15q Alliance
- American Cleft Palate-Craniofacial Association
- 5P-Society
- American Society for Reproductive Medicine
- Turner Syndrome Society of the United States
- American Bronchoesophagological Association
- American Speech-Language-Hearing Association
- National Hemophilia Foundation
- Moebius Syndrome Foundation
- PRISMS (Parents & Researchers Interested in Smith-Magenis Syndrome)
- Trisomy 9 International Parent Support (9TIPS)
- Apert Syndrome Support Group
- Mental Health America
- Weill Cornell Medical College - Division of Pediatric Genetics
- American Leprosy Missions
- Little People of America, Inc.
- Bloom's Syndrome Registry
- National Kidney Foundation
- National Federation of the Blind
- National Mental Health Consumers' Self-Help Clearinghouse
- Learning Disabilities Association of America
- American Childhood Cancer Organization
- National Down Syndrome Society
- Chromosome Disorder Outreach, Inc.
- National Center for Learning Disabilities
- Sjogren's Syndrome Foundation, Inc.
- Gallaudet University Genetics Program
- Virginia Commonwealth University School of Medicine - Dept. Human & Molecular Genetics
- Alexander Graham Bell Association for the Deaf and Hard of Hearing
- Cure SMA
- American Sleep Association
- Ataxia Telangiectasia Children's Project, Inc (A-T)
- Weaver Syndrome Network
- HRSA Information Center
- Angelman Syndrome Foundation, Inc.
- Lupus Foundation of America, Inc.
- CHARGE Syndrome Foundation, Inc.
- United Leukodystrophy Foundation
- Acoustic Neuroma Association
- Marfan Foundation
- Sickle Cell Disease Association of America, Inc.
- Michigan State University Human Genetics Program
- Center for Medical Genetics
- ARCH National Respite Network
- National Association for Parents of Children with Visual Impairments (NAPVI)
- American Liver Foundation
- National Association of Hospital Hospitality Houses, Inc.
- Immune Deficiency Foundation
- TMJ Association, Ltd.
- International Joseph Disease Foundation, Inc.
- Narcolepsy Network, Inc.
- Norrie Disease Association
- Cooley's Anemia Foundation, Inc.
- Taking Action Against Language Disorders for Kids (TALK)
- Hydrocephalus Association
- Children's Brain Diseases Foundation
- National Federation of Families for Children's Mental Health
- Mario Negri Institute for Pharmacological Research
- Smell and Taste Center
- Rocky Mountain Disability & Business Technical Assistance Center
- National Down Syndrome Congress
- Mobility International USA
- Blood & Marrow Transplant Information Network
- David G. Jagelman Inherited Colorectal Cancer Registries
- Children’s PKU Network
- Friends of Disabled Adults and Children, Too! Inc.
- Family Resource Center on Disabilities
- New England Regional Genetics Group
- Hereditary Disease Foundation, Inc.
- Chronic Granulomatous Disease Association, Inc.
- ALS Association
- AmeriFace
- Batten Disease Support and Research Association
- Congenital CMV Disease Research Clinic & Registry
- National Sleep Foundation
- International Children's Anophthalmia Network (ICAN)
- National Scoliosis Foundation
- Familial GI Cancer Registry
- VOR
- Obesity Medicine Association
- Hermansky-Pudlak Syndrome Network, Inc.
- National MPS Society, Inc.
- American Autoimmune & Related Diseases Association, Inc.
- MAB Community Services
- Fanconi Anemia Research Fund, Inc.
- Interstitial Cystitis Association
- Neurofibromatosis Network
- National Fragile X Foundation
- National Niemann-Pick Disease Foundation, Inc.
- National Society of Genetic Counselors
- Metatropic Dysplasia Dwarf Registry
- National Spasmodic Torticollis Association
- FRAXA Research Foundation
- Association of Gastrointestinal Motility Disorders, Inc. (AGMD)
- International Rett Syndrome Foundation
- Klippel-Trenaunay Support Group
- Pulmonary Hypertension Association
- Alagille Syndrome Alliance
- The Ehlers-Danlos Society
- National Headache Foundation
- Foundation for Nager and Miller Syndromes
- World Life Foundation
- Mucolipidosis Type IV Foundation, Inc. (ML4Fdn)
- Lactic Acidosis Support Trust
- Myotubular Myopathy Resource Group
- Piedmont Health Services and Sickle Cell Agency
- United Mitochondrial Disease Foundation
- Sturge-Weber Foundation
- Schepens Eye Research Institute
- FSHD Society
- Spina Bifida Association
- Spondylitis Association of America
- National Organization for Rare Disorders, Inc.
- National Women's Health Resource Center
- Any Baby Can of San Antonio
- CCHS Family Network (Congenital Central Hypoventilation Syndrome)
- American Juvenile Arthritis Organization
- Scleroderma Foundation
- Cyclic Vomiting Syndrome Association
- Scleroderma Research Foundation
- Osteogenesis Imperfecta Foundation
- American Kidney Fund, Inc.
- Rubinstein-Taybi Parent Group
- National PKU News
- Graves' Disease & Thyroid Foundation
- National Ataxia Foundation
- National Association for Pseudoxanthoma Elasticum (NAPE)
- National Temporal Bone, Hearing & Balance Pathology Resource Registry
- American Chronic Pain Association
- Coffin-Lowry Syndrome Foundation
- Let's Face It
- Cornelia de Lange Syndrome Foundation, Inc.
- Hearing Health Foundation
- National Hydrocephalus Foundation
- The Arc
- Twin to Twin Transfusion Syndrome Foundation
- National Vulvodynia Association
- National Gaucher Foundation
- American Association of Kidney Patients
- Malignant Hyperthermia Association of the United States
- American Tinnitus Association
- Foundation for Ichthyosis & Related Skin Types
- Recurrent Respiratory Papillomatosis Foundation
- FACES: The National Craniofacial Association
- National Multiple Sclerosis Society
- Share and Care Cockayne Syndrome Network, Inc.
- Lowe Syndrome Association, Inc.
- Shwachman-Diamond Syndrome Foundation
- National Center for Chromosome Inversions
- National Association for Continence
- Ambiguous Genitalia Support Network
- March of Dimes
- Nevus Network
- Support Organization for Trisomy 18, 13, and Related Disorders
- American Brain Tumor Association
- Dystonia Medical Research Foundation
- National Tay-Sachs and Allied Diseases Association, Inc.
- National Psoriasis Foundation
- Vaincre Les Maladies Lysosomales
- Foundation Fighting Blindness
- National Adrenal Diseases Foundation
- Restless Legs Syndrome Foundation
- Charcot-Marie-Tooth Association
- Reflex Sympathetic Dystrophy Syndrome Association (RSDSA)
- National Organization for Albinism and Hypopigmentation (NOAH)
- Wilson Disease Association
- Cystic Fibrosis Foundation
- National Rehabilitation Information Center
- Joubert Syndrome and Related Disorders Foundation
- Prader-Willi Syndrome Association (USA)
- International Essential Tremor Foundation
- Vasculitis Foundation
- National Alopecia Areata Foundation
- National Amputation Foundation, Inc.
- International FOP Association
- National Prune Belly Syndrome Network
- Leukemia & Lymphoma Society
- National Reye's Syndrome Foundation, Inc.
- First Candle-SIDS Alliance
- National Organization on Fetal Alcohol Syndrome
- Smith-Lemli-Opitz/RSH Foundation
- Debra of America, Inc. (Dystrophic Epidermolysis Bullosa)
- Benign Essential Blepharospasm Research Foundation, Inc.
- Children’s Tumor Foundation
- Parkinson's Disease Foundation, Inc.
- International OCD Foundation
- National Lymphedema Network, Inc.
- Children’s Craniofacial Association
- National Spasmodic Dysphonia Association
- Association for Glycogen Storage Disease
- National Foundation for Ectodermal Dysplasias
- MAGIC Foundation
- International Long QT Syndrome Registry
- Kennedy Krieger Institute
- Myositis Association
- Freeman-Sheldon Research Group, Inc.
- Oxalosis and Hyperoxaluria Foundation
- Histiocytosis Association, Inc.
- Human Growth Foundation
- Huntington's Disease Society of America
- GBS/CIDP Foundation International
- Familial Dysautonomia Foundation
- American Behçet’s Disease Association
- Aplastic Anemia & MDS International Foundation, Inc.
- American Porphyria Foundation
- Myasthenia Gravis Foundation of America, Inc.
- Tuberous Sclerosis Alliance
- National Urea Cycle Disorders Foundation
- Metabolic Support UK
- Tourette Association of America
- American Syringomyelia & Chiari Alliance Project, Inc.
- TNA – The Facial Pain Association
- VHL Alliance
- Williams Syndrome Association
- Vestibular Disorders Association (VEDA)
- Hypoparathyroidism Association, Inc.
- Prion Alliance
- Dent Disease Foundation
- MECP2 Duplication UK
- Ring20 Research and Support UK
- Eye on Vision Foundation
- Cluster Headache Support Group, Inc.
- Castleman Disease Collaborative Network
- Turner Syndrome Foundation
- FH Foundation
- Cat Eye Syndrome International
- PTEN Hamartoma Tumor Syndrome Foundation
- U.R. Our Hope
- Sneddon's Foundation
- Wake Up Narcolepsy, Inc
- MDS Foundation, Inc.
- International Eosinophil Society, Inc
- Rare Bleeding Disorders Database (RBDD)
- Cohen Syndrome Association
- MHE Research Foundation
- Progressive Familial Intrahepatic Cholestasis Web Group
- PKS Kids
- Fibrosing Mediastinitis Informational Website
- Costello Syndrome Family Network
- Oklahoma Disability Law Center, Inc.
- American College of Physicians - Internal Medicine
- Angel Flight East
- National Marrow Donor Program
- Make Early Diagnosis To Prevent Early Death
- PXE International, Inc.
- Washington State Department of Social and Health Services
- NIH/National Center for Complementary and Alternative Medicine
- Center for Universal Design
- Urology Care Foundation
- National Research Center for Career and Technical Education
- Chromosome 9pminus Network
- 4P- Support Group, Inc.
- Crouzon Support Network, A Program of AmeriFace
Rare Diseases
- STXBP1 Disorders
- MEF2C Deficiency
- MN1 C-Terminal Truncation Syndrome
- GNE Myopathy
- Aromatic L-Amino Acid Decarboxylase Deficiency
- Superficial Siderosis
- Palmoplantar Pustulosis
- Systemic Scleroderma
- Takotsubo Cardiomyopathy
- Miller Fisher Syndrome
- Thyroid Eye Disease
- Acute Promyelocytic Leukemia
- Meningioma
- Primary Central Nervous System Lymphoma
- Generalized Arterial Calcification of Infancy
- Autosomal Recessive Hypophosphatemic Rickets Type 2
- Renal Medullary Carcinoma
- Complete DiGeorge Syndrome
- Cerebral Folate Deficiency
- Craniopharyngioma
- Glioma
- Spinal Muscular Atrophy with Respiratory Distress
- Spastic Paraplegia 47
- AP-4-Associated Hereditary Spastic Paraplegia (AP-4-HSP)
- Spastic Paraplegia 52
- Spastic Paraplegia 51
- Spastic Paraplegia 50
- KCNB1 Encephalopathy
- Gestational Trophoblastic Disease
- Pediatric Non-Small Cell Lung Cancer
- Amniotic Fluid Embolism
- Esophageal Cancer
- Follicular Lymphoma
- ADCY5-Related Dyskinesia
- Bohring-Opitz Syndrome
- Deoxyhypusine Synthase Disorder
- Hemiplegic Migraine
- Primary Mitochondrial Myopathies
- Potter Syndrome
- Pediatric Crohn’s Disease
- Protein S Deficiency
- Stomach Cancer
- COPA Syndrome
- KIF1A-Related Disorder
- Neurotrophic Keratitis
- Ovarian Cancer
- Primary Distal Renal Tubular Acidosis
- Argininie: Glycine Amidinotransferase Deficiency
- Guanidinoacetate Methyltransferase Deficiency
- Creatine Transporter Deficiency
- ADNP Syndrome
- SLC6A1 Epileptic Encephalopathy
- COL4A1/A2-Related Disorders
- Testicular Cancer
- Small Cell Lung Cancer
- Riboflavin Transporter Deficiency
- Kufor Rakeb Syndrome
- SETBP1 Disorder
- Median Arcuate Ligament Syndrome
- Focal Segmental Glomerulosclerosis
- Superior Mesenteric Artery Syndrome
- KAT6A Syndrome
- Soft Tissue Sarcoma
- Multifocal Motor Neuropathy
- Dup15q Syndrome
- Post-Transplant Lymphoproliferative Disease
- Cerebral Creatine Deficiency Syndromes
- Pure Autonomic Failure
- Merkel Cell Carcinoma
- Mucormycosis
- Factor X Deficiency
- TANGO2-Related Metabolic Encephalopathy and Arrhythmias
- Familial Platelet Disorder with Associated Myeloid Malignancy
- Hereditary Orotic Aciduria
- Leukoencephalopathy with Brain Stem and Spinal Cord Involvement and Lactate Elevation
- Osteosarcoma
- Autoinflammation with Infantile Enterocolitis
- Hemophagocytic Lymphohistiocytosis
- Primary Hyperparathyroidism
- Hereditary Breast and Ovarian Cancer Syndrome
- Hashimoto Encephalopathy
- Gerstmann-Sträussler-Scheinker Disease
- Ocular Melanoma
- Laurence-Moon Syndrome
- Goblet Cell Carcinoid
- Appendiceal Cancer and Tumors
- Visual Snow Syndrome
- Urachal Cancer
- PHACE Syndrome
- Antisynthetase Syndrome
- Liposarcoma
- Fibrolamellar Carcinoma
- Pneumocystis Pneumonia
- Clostridial Myonecrosis
- Pitt-Hopkins Syndrome
- Fatal Familial Insomnia
- NGLY1 Deficiency
- Hepatopulmonary Syndrome
- USP7-Related Diseases
- Tenosynovial Giant Cell Tumor
- Spondylothoracic Dysplasia
- SLC13A5 Epileptic Encephalopathy
- Li-Fraumeni Syndrome
- RYR-1-Related Diseases
- Sitosterolemia
- Retinal Vasculopathy with Cerebral Leukoencephalopathy and Systemic Manifestations
- Bosma Arhinia Microphthalmia Syndrome
- Shprintzen Goldberg Syndrome
- Hepatocellular Carcinoma
- CARD9 Deficiency
- Acute Myeloid Leukemia
- Succinic Semialdehyde Dehydrogenase Deficiency
- Hereditary Sensory and Autonomic Neuropathy Type 1E
- Alpha Thalassemia
- Spontaneous Intracranial Hypotension
- Protein C Deficiency
- Warburg Micro Syndrome
- Autoimmune Hepatitis
- KCNK9 Imprinting Syndrome
- Leishmaniasis
- Acid Sphingomyelinase Deficiency
- Congenital Myasthenic Syndromes
- KCNQ2 Encephalopathy
- Superior Semicircular Canal Dehiscence
- Snyder-Robinson Syndrome
- Hallermann Streiff Syndrome
- Nontuberculous Mycobacterial Lung Disease
- Idiopathic Subglottic Stenosis
- Sepiapterin Reductase Deficiency
- Gitelman Syndrome
- Andersen-Tawil Syndrome
- Myocarditis
- Recessive Multiple Epiphyseal Dysplasia
- SYNGAP1-related NSID
- Necrotizing Enterocolitis
- ATR-16 Syndrome
- Zollinger-Ellison Syndrome
- Zellweger Spectrum Disorders
- Yunis Varon Syndrome
- Yellow Nail syndrome
- Yellow Fever
- Yaws
- XYY Syndrome
- Xeroderma Pigmentosum
- X-Linked Protoporphyria
- X-linked Opitz G/BBB Syndrome
- X-Linked Myotubular Myopathy
- X-Linked Myopathy with Excessive Autophagy
- X linked Lymphoproliferative Syndrome
- X-linked Retinoschisis
- Wyburn-Mason Syndrome
- Wolman Disease
- Wolfram Syndrome
- Wolff Parkinson White Syndrome
- Wolf-Hirschhorn Syndrome
- WNT4 Deficiency
- Winchester Syndrome
- Wilson Disease
- Wilms' Tumor
- Williams Syndrome
- Wildervanck Syndrome
- Wiedemann Rautenstrauch Syndrome
- Wieacker Syndrome
- Whipple Disease
- WHIM Syndrome
- West Syndrome
- West Nile Encephalitis
- Wernicke-Korsakoff Syndrome
- Werner Syndrome
- Werdnig-Hoffmann Disease
- Weismann Netter Stuhl Syndrome
- Weill Marchesani Syndrome
- Weil Syndrome
- Granulomatosis with Polyangiitis
- Weaver Syndrome
- WAS Related Disorders
- Warm Autoimmune Hemolytic Anemia
- Wandering Spleen
- Walker Warburg Syndrome
- Waldenström's Macroglobulinemia
- WAGR Syndrome/11p Deletion Syndrome
- Waardenburg Syndrome
- Von Willebrand Disease
- Von Hippel-Lindau Disease
- Vogt-Koyanagi-Harada Disease
- Very Long Chain Acyl CoA Dehydrogenase Deficiency (LCAD)
- Vernal Keratonconjunctivitis
- Ventricular Septal Defects
- Cutaneous Vasculitis
- Vasculitis
- Vascular Malformations of the Brain
- Variegate Porphyria
- Valinemia
- VACTERL with Hydrocephalus
- VACTERL Association
- Uterine Leiomyosarcoma
- Usher Syndrome
- Urticaria, Physical
- Urticaria, Papular
- Urticaria, Cold
- Ulcerative Colitis
- Tyrosinemia Type 1
- Tyrosine Hydroxylase Deficiency
- STEC Hemolytic Uremic Syndrome
- Typhoid
- Twin-Twin Transfusion Syndrome
- Turner Syndrome
- Turcot Syndrome
- Tumor Necrosis Factor Receptor-Associated Periodic Syndrome
- Tularemia
- Tuberous Sclerosis
- Tuberculosis
- Truncus Arteriosus
- Tropical Sprue
- Trisomy X
- Trisomy 18
- Trisomy 13 Syndrome
- Trismus-Pseudocamptodactyly Syndrome
- Triploidy
- Triosephosphate Isomerase Deficiency
- Trimethylaminuria
- Trigeminal Neuralgia
- Trichotillomania
- Trichorhinophalangeal Syndrome Type III
- Trichorhinophalangeal Syndrome Type II
- Trichorhinophalangeal Syndrome Type I
- Tricho Dento Osseous Syndrome
- Treacher Collins Syndrome
- Transverse Myelitis
- Toxic Shock Syndrome
- Staphylococcal Scalded Skin Syndrome
- Townes Brocks Syndrome
- TORCH Syndrome
- Tooth and Nail Syndrome
- Tongue, Hairy
- Tongue Cancer
- Tolosa Hunt Syndrome
- Tinnitus
- Timothy Syndrome
- Tietze Syndrome
- Thyroid Cancer
- Thrombotic Thrombocytopenic Purpura
- Thrombocytopenia Absent Radius Syndrome
- Three M Syndrome
- Thoracic Outlet Syndrome
- Beta Thalassemia
- Tetralogy of Fallot
- Tetrahydrobiopterin Deficiency
- Tethered Cord Syndrome
- Tay Sachs Disease
- Tarsal Tunnel Syndrome
- Tarlov Cysts
- Tardive Dyskinesia
- Tangier Disease
- Systemic Capillary Leak Syndrome
- Syringomyelia
- Syringobulbia
- Congenital Syphilis
- Syphilis, Acquired
- Sydenham Chorea
- Swyer syndrome
- Sweet Syndrome
- Sutton Disease II
- Susac Syndrome
- Sudden Unexplained Death in Childhood
- Sudden Infant Death Syndrome
- Subacute Sclerosing Panencephalitis
- Stuve-Wiedemann Syndrome
- Sturge Weber Syndrome
- Stiff Person Syndrome
- Stickler Syndrome
- Stevens-Johnson Syndrome and Toxic Epidermal Necrolysis
- Status Epilepticus
- Sprengel Deformity
- Sporadic Porencephaly
- Sporadic Inclusion Body Myositis
- Spondyloepiphyseal Dysplasia, Congenital
- Spondyloepiphyseal Dysplasia Tarda
- Split Hand/Split Foot Malformation
- Spinocerebellar Ataxia with Axonal Neuropathy
- Spinal Muscular Atrophy
- Spina Bifida
- Sotos Syndrome
- Sneddon Syndrome
- Smith Magenis Syndrome
- Smith Lemli Opitz Syndrome
- Smallpox
- Mucopolysaccharidosis Type VII
- Sirenomelia
- Sinonasal Undifferentiated Carcinoma
- Singleton Merten syndrome
- Simpson-Golabi-Behmel Syndrome
- Simple Pulmonary Eosinophilia
- Simian B Virus Infection
- Sickle Cell Disease
- Sialidosis
- Sialadenitis
- Shwachman Diamond Syndrome
- SHORT Syndrome
- Short Chain Acyl CoA Dehydrogenase Deficiency
- Short Bowel Syndrome
- Sheehan Syndrome
- Severe Combined Immunodeficiency
- Severe Chronic Neutropenia
- Setleis Syndrome
- Sennetsu Fever
- Segawa Syndrome
- Seckel Syndrome
- Scott Craniodigital Syndrome
- Mesenteric Panniculitis
- Scleroderma
- Schwartz Jampel Syndrome
- Schnitzler Syndrome
- Schinzel Syndrome
- Schinzel Giedion Syndrome
- Schindler disease
- Nevus Sebaceus Syndrome
- Schimke Immuno-Osseous Dysplasia
- Classic Infantile CLN1 Disease
- Sandhoff Disease
- Sakati Syndrome
- Saethre Chotzen Syndrome
- Sacrococcygeal Teratoma
- Bannayan-Riley-Ruvalcaba Syndrome
- Russell-Silver Syndrome
- Rubinstein Taybi Syndrome
- Rubella, Congenital
- Rubella
- Roussy Lévy Syndrome
- Rothmund-Thomson Syndrome
- Rosenberg Chutorian Syndrome
- Rosai-Dorfman Disease
- Long QT Syndrome
- Rocky Mountain Spotted Fever
- Robinow Syndrome
- Roberts Syndrome
- Ring Chromosome 4
- Rieger Syndrome
- Vitamin D Deficiency Rickets
- Rheumatic Fever
- Reye Syndrome
- Rett Syndrome
- Retroperitoneal Fibrosis
- Retinoschisis
- Retinopathy of Prematurity
- Retinoblastoma
- Retinitis Pigmentosa
- Restless Legs Syndrome
- Respiratory Distress Syndrome, Infant
- Renal Glycosuria
- Renal Cell Carcinoma
- Renal Agenesis, Bilateral
- Relapsing Polychondritis
- Refsum Disease
- Refractory Celiac Disease
- Complex Regional Pain Syndrome
- Recurrent Respiratory Papillomatosis
- Reactive Arthritis
- Rasmussen Encephalitis
- Rapid-onset Obesity with Hypothalamic Dysfunction, Hypoventilation, and Autonomic Dysregulation
- Ramsay Hunt Syndrome
- Radiation Sickness
- Rabson-Mendenhall Syndrome
- Rabies
- Q fever
- Pyruvate Kinase Deficiency
- Pyruvate Dehydrogenase Complex Deficiency
- Pyruvate Carboxylase Deficiency
- Pyridoxine-Dependent Epilepsy
- Pyoderma Gangrenosum
- Pycnodysostosis
- Pure Red Cell Aplasia, Acquired
- Pulmonary Arterial Hypertension
- Pulmonary Alveolar Proteinosis
- Pterygium Syndrome, Multiple
- PTEN Hamartoma Tumor Syndrome
- Psittacosis
- Pseudoxanthoma Elasticum
- Pseudomyxoma Peritonei
- Pseudohypoparathyroidism
- Pseudocholinesterase Deficiency
- Pseudoachondroplasia
- Pseudo Hurler Polydystrophy
- Prune Belly Syndrome
- Proteus Syndrome
- Prolactinoma
- Erythrokeratoderma
- Progressive Supranuclear Palsy
- Progressive Osseous Heteroplasia
- Progressive Myoclonus Epilepsy
- Progressive Multifocal Leukoencephalopathy
- Proctitis
- Primary Visual Agnosia
- Primary Sclerosing Cholangitis
- Primary Orthostatic Tremor
- Primary Myelofibrosis
- Primary Lateral Sclerosis
- Primary Intestinal Lymphangiectasia
- Primary Hyperoxaluria
- Primary Gastric Lymphoma
- Primary Craniosynostosis
- Primary Ciliary Dyskinesia
- Primary Biliary Cholangitis
- Precocious Puberty
- Prader-Willi Syndrome
- Posterior Uveitis
- Post Polio Syndrome
- Congenital Erythropoietic Porphyria
- Porphyria Cutanea Tarda
- Porphyria
- Pontocerebellar Hypoplasia
- Pompe Disease
- Polymyositis and Necrotizing Myopathy
- Polymyalgia Rheumatica
- Polymorphous Low-Grade Adenocarcinoma
- Adult Polyglucosan Body Disease
- Polycythemia Vera
- Polycystic Liver Disease
- Polyarteritis Nodosa
- Poland Syndrome
- POEMS Syndrome
- PMM2-CDG
- Pleuropulmonary Blastoma
- Plague
- Pityriasis Rubra Pilaris
- Pityriasis Rosea
- Pinta
- Pierre Robin Sequence
- Frontotemporal Degeneration
- Phosphoglycerate Kinase Deficiency
- Pheochromocytoma
- Phenylketonuria
- Phelan-McDermid Syndrome
- Pfeiffer Syndrome
- Peutz Jeghers Syndrome
- Pertussis
- Perniosis
- PEPCK Deficiency
- Pentalogy of Cantrell
- Penta X Syndrome
- Pemphigus and Pemphigoid
- Pelizaeus-Merzbacher Disease
- Peeling Skin Syndrome
- Pediatric Cardiomyopathy
- Parsonage Turner Syndrome
- Pars Planitis
- Parry Romberg Syndrome
- Paroxysmal Nocturnal Hemoglobinuria
- Paroxysmal Cold Hemoglobinuria
- Paraneoplastic Neurologic Syndromes
- Paramyotonia Congenita
- Paracoccidioidomycosis
- Papillon Lefèvre Syndrome
- Papillitis
- Pantothenate Kinase-Associated Neurodegeneration
- Panniculitis, Idiopathic Nodular
- Pancreatic Neuroendocrine Neoplasms (pNENs)
- Pallister W Syndrome
- Pallister Killian Mosaic Syndrome
- Pallister-Hall Syndrome
- Paget's Disease of the Breast
- Paget's Disease
- Pachyonychia Congenita
- Pachydermoperiostosis
- Ovotesticular Disorder of Sex Development
- Otopalatodigital Syndrome Type I and II
- Osteopetrosis
- Osteonecrosis
- Osteomyelitis
- Osteogenesis Imperfecta
- OSMED, Homozygous
- OSMED, Heterozygous
- Orthostatic Hypotension
- Orocraniodigital Syndrome
- Ornithine Transcarbamylase Deficiency
- Oral-Facial-Digital Syndrome
- Optic Nerve Hypoplasia
- Opsoclonus-Myoclonus Syndrome
- Ollier Disease
- Olivopontocerebellar Atrophy
- Ogilvie syndrome
- Oculopharyngeal Muscular Dystrophy
- Oculocutaneous Albinism
- Oculocerebrocutaneous Syndrome
- Oculocerebral Syndrome with Hypopigmentation
- Oculo-Dento-Digital Dysplasia
- Oculo-Auriculo-Vertebral Spectrum
- Ocular Motor Apraxia, Cogan Type
- Ocular Albinism
- Urofacial Syndrome
- Norrie Disease
- Noonan Syndrome
- Nonketotic Hyperglycinemia
- Non-24-Hour Sleep-Wake Disorder
- Nocardiosis
- Niemann Pick Disease Type C
- NORSE (New Onset Refractory Status Epilepticus) and FIRES (Febrile Infection-Related Epilepsy Syndrome)
- Nevoid Basal Cell Carcinoma Syndrome
- Neuropathy, Congenital Hypomyelination
- Neuromyelitis Optica Spectrum Disorder
- Neuroleptic Malignant Syndrome
- Neurofibromatosis 2
- Neurofibromatosis 1
- Neuroacanthocytosis
- Neu Laxova Syndrome
- Nephrogenic Systemic Fibrosis
- Nephrogenic Diabetes Insipidus
- Neonatal-Onset Multisystem Inflammatory Disease
- Neonatal Lupus
- Neonatal Hemochromatosis
- Nemaline Myopathy
- Nelson Syndrome
- Necrotizing Fasciitis
- Narcolepsy
- Nance-Horan Syndrome
- Nail Patella Syndrome
- Nager Syndrome
- N-Acetylglutamate Synthetase Deficiency
- Myotonia Congenita
- Myopathy, Scapuloperoneal
- Myopathy, Myofibrillar
- Congenital Myopathy
- Myhre Syndrome
- Myelodysplastic Syndromes
- Mycosis Fungoides
- Myasthenia Gravis
- Mutism, Selective
- Muscular Dystrophy, Becker
- Mumps
- Mulvihill Smith Syndrome
- Multiple System Atrophy
- Multiple Sulfatase Deficiency
- Multiple Sclerosis
- Multiple Myeloma
- Dominant Multiple Epiphyseal Dysplasia
- Multiple Endocrine Neoplasia Type 2
- Multiple Endocrine Neoplasia Type 1
- Mulibrey Nanism
- Mucous Membrane Pemphigoid
- Mucopolysaccharidosis Type III
- Mucopolysaccharidosis Type I
- Mucopolysaccharidoses
- Mucolipidosis IV
- Muckle-Wells Syndrome
- Mucha Habermann Disease
- Moyamoya Disease
- Mowat-Wilson Syndrome
- Mucopolysaccharidosis IV
- Monilethrix
- Moebius Syndrome
- Mixed Cryoglobulinemia
- Mixed Connective Tissue Disease (MCTD)
- Mitral Valve Prolapse Syndrome
- Mitochondrial Neurogastrointestinal Encephalopathy
- Miller Syndrome
- Mikulicz Syndrome
- Microvillus Inclusion Disease
- Metatropic Dysplasia I
- Metaphyseal Chondrodysplasia, Schmid Type
- Mesothelioma
- Mesenchymal Chondrosarcoma
- MERRF Syndrome
- Menkes Disease
- Meningococcemia
- Meningococcal Meningitis
- Meningitis, Tuberculous
- Meningitis, Bacterial
- Meningitis
- Ménière's Disease
- Menetrier Disease
- Melorheostosis
- Melnick Needles Syndrome
- Melkersson-Rosenthal Syndrome
- Meleda Disease
- MELAS Syndrome
- Melanoma, Malignant
- Meige Syndrome
- Megalocornea Intellectual Disability Syndrome
- Megalencephaly-Capillary Malformation
- Medulloblastoma
- Medullary Sponge Kidney
- Medium Chain Acyl CoA Dehydrogenase Deficiency
- MECP2 Duplication Syndrome
- Meckel Syndrome
- Measles
- MDR3 Deficiency
- MCT8-Specific Thyroid Hormone Cell Transporter Deficiency
- McKusick Type Metaphyseal Chondrodysplasia
- McCune Albright Syndrome
- Mayer-Rokitansky-Küster-Hauser Syndrome
- May Hegglin Anomaly
- Maxillofacial Dysostosis
- Maternally Inherited Leigh Syndrome and NARP Syndrome
- Mastocytosis
- Marshall Syndrome
- Marshall Smith Syndrome
- Maroteaux Lamy Syndrome
- Marinesco-Sjögren Syndrome
- Marfan Syndrome
- Marden Walker Syndrome
- Marcus Gunn Phenomenon
- Maple Syrup Urine Disease
- Mantle Cell Lymphoma
- Mandibuloacral Dysplasia
- Mallory Weiss Syndrome
- Malignant Hyperthermia
- Malaria
- Mal de Debarquement
- Maffucci Syndrome
- Madelung's Disease
- Macroglossia
- Machado-Joseph Disease
- Lysosomal Storage Disorders
- Lysosomal Free Sialic Acid Storage Disorders
- Lymphomatoid Granulomatosis
- Lymphocytic Infiltrate of Jessner
- Lymphedema-Distichiasis Syndrome
- Lymphatic Malformations
- Lymphangioleiomyomatosis
- Lowe syndrome
- Low Gamma-GT Familial Intrahepatic Cholestasis
- Senior Løken Syndrome
- Locked In Syndrome
- Listeriosis
- Lissencephaly
- Limb-Girdle Muscular Dystrophies
- Lichen Sclerosus
- Lichen Planus
- Levy-Yeboa Syndrome
- Metachromatic Leukodystrophy
- Leukodystrophy, Krabbe's
- Leukodystrophy
- Leukocyte Adhesion Deficiency Syndromes
- Lesch Nyhan Syndrome
- Leri Pleonosteosis
- Leptospirosis
- Leprosy
- Leprechaunism
- Noonan Syndrome with Multiple Lentigines
- Lenz Microphthalmia Syndrome
- Lennox-Gastaut Syndrome
- Leiomyosarcoma, Inferior Vena Cava
- Leiomyosarcoma
- Leigh Syndrome
- Legionnaires' Disease
- Legg Calvé Perthes Disease
- Leber Hereditary Optic Neuropathy
- Leber Congenital Amaurosis
- Laryngeal Dystonia
- Larsen Syndrome
- Langerhans Cell Histiocytosis
- Landau Kleffner Syndrome
- Lambert-Eaton Myasthenic Syndrome
- LADD syndrome
- Laband Syndrome
- L1 Syndrome
- Kugelberg Welander Syndrome
- Adult Neuronal Ceroid Lipofuscinosis
- Kohler Disease
- Kniest Dysplasia
- Klüver-Bucy Syndrome
- Klippel-Trenaunay Syndrome
- Klippel-Feil Syndrome
- 47,XXY (Klinefelter Syndrome)
- Kleine-Levin Syndrome
- Kikuchi's Disease
- Kienböck Disease
- Kernicterus
- Keratosis, Seborrheic
- Keratosis Follicularis
- Keratomalacia
- Keratoconus
- Keratitis Ichthyosis Deafness Syndrome
- Kenny-Caffey Syndrome
- Kennedy Disease
- Kearns Sayre Syndrome
- KBG Syndrome
- Kawasaki Disease
- Kasabach-Merritt Phenomenon
- Kallmann Syndrome
- Kabuki Syndrome
- Juvenile Pilocytic Astrocytoma
- Juvenile Myelomonocytic Leukemia
- Juvenile Hemochromatosis
- Jumping Frenchmen of Maine
- Juberg-Marsidi Syndrome
- Joubert Syndrome
- Johanson-Blizzard Syndrome
- Jervell and Lange-Nielsen Syndrome
- Jejunal Atresia
- Spondylocostal Dysplasia
- Jansen Type Metaphyseal Chondrodysplasia
- Jackson-Weiss Syndrome
- Ivemark Syndrome
- IRF6-Related Disorders
- PLA2G6-Associated Neurodegeneration
- Infantile Myofibromatosis
- Incontinentia Pigmenti
- Imperforate Anus
- IgA Nephropathy
- Immune Thrombocytopenia
- Idiopathic Pulmonary Fibrosis
- Neonatal Cholestasis
- Idiopathic Intracranial Hypertension
- Ichthyosis, X Linked
- Trichothiodystrophy
- Sjögren-Larsson Syndrome
- Ichthyosis, Netherton Syndrome
- Lamellar Ichthyosis
- Keratosis Follicularis Spinulosa Decalvans
- Harlequin Ichthyosis
- Ichthyosis, Erythrokeratolysis Hiemalis
- Ichthyosis, CHILD Syndrome
- Chanarin-Dorfman Syndrome
- Ichthyosis Vulgaris
- Ichthyosis Hystrix, Curth Macklin Type
- Ichthyosis
- I Cell Disease
- Hypothalamic Hamartoma
- Hypoplastic Left Heart Syndrome
- Hypophosphatasia
- Hypoparathyroidism
- Hypomelanosis of Ito
- Hypokalemia
- Hypohidrotic Ectodermal Dysplasia
- Hypochondroplasia
- Hyperprolinemia Type II
- Hyperprolinemia Type I
- Hyperostosis Frontalis Interna
- Hyperlipoproteinemia Type III
- Hyperhidrosis, Primary
- Hyperferritinemia Cataract Syndrome
- Hyperemesis Gravidarum
- Hyperekplexia
- Hyper IgM Syndromes
- Mevalonate Kinase Deficiency
- Hydrocephalus
- Hydranencephaly
- Hutchinson-Gilford Progeria Syndrome
- Huntington's Disease
- Mucopolysaccharidosis Type II
- Human Monocytic Ehrlichiosis (HME)
- Human HOXA1 Syndromes
- Human Granulocytic Ehrlichiosis (HGE)
- HTLV Type I and Type II
- Horner's Syndrome
- Homocystinuria due to Cystathionine Beta-Synthase Deficiency
- Holt Oram Syndrome
- Holoprosencephaly
- Hodgkin's Disease
- Histidinemia
- Hirschsprung Disease
- Hidradenitis Suppurativa
- Hiccups, Chronic
- Glycogen Storage Disease Type VI
- Herpes, Neonatal
- Hermansky Pudlak Syndrome
- Hereditary Spastic Paraplegia
- Hereditary Sensory Neuropathy Type I
- Hereditary Sensory and Autonomic Neuropathy Type IV
- Hereditary Sensory and Autonomic Neuropathy Type II
- Hereditary Neuralgic Amyotrophy
- Hereditary Multiple Osteochondromas
- Hereditary Lymphedema
- Hereditary Leiomyomatosis and Renal Cell Carcinoma
- Hereditary Hyperphosphatasia
- Hereditary Hemorrhagic Telangiectasia
- Hereditary Coproporphyria
- Hepatorenal Syndrome
- Hepatoerythropoietic Porphyria
- Congenital Hepatic Fibrosis
- Hepatic Encephalopathy
- Henoch-Schönlein Purpura
- Hemophilia B
- Hemophilia A
- Hemimegalencephaly
- Heavy Metal Poisoning
- Congenital Heart Block
- Hartnup Disease
- Hantavirus Pulmonary Syndrome
- Hanhart Syndrome
- Hajdu Cheney Syndrome
- Hairy Cell Leukemia
- Haim-Munk Syndrome
- Hailey-Hailey Disease
- Guillain-Barré Syndrome
- Growth Hormone Insensitivity
- Growth Hormone Deficiency
- Grover's Disease
- Greig Cephalopolysyndactyly Syndrome
- Graves' Disease
- Granuloma Annulare
- Graft versus Host Disease
- Gottron Syndrome
- Gorlin-Chaudhry-Moss Syndrome
- Gorham-Stout Disease
- Gordon Syndrome
- Goodpasture Syndrome
- Glycogen Storage Disease Type 7
- Glycogen Storage Disease Type V
- Glycogen Storage Disease Type IX
- Glycogen Storage Disease Type I
- Glutaric Aciduria Type II
- Glutaric Aciduria Type I
- Glucose-Galactose Malabsorption
- Glucose-6-Phosphate Dehydrogenase Deficiency
- Glucose Transporter Type 1 Deficiency Syndrome
- Glioblastoma
- Glanzmann Thrombasthenia
- Gilbert Syndrome
- Giant Congenital Melanocytic Nevus
- Giant Cell Myocarditis
- Giant Axonal Neuropathy
- Gianotti Crosti Syndrome
- Gerstmann Syndrome
- Geographic Tongue
- General Myoclonus
- Gaucher Disease
- Gastroschisis
- Gastroparesis
- Gastrointestinal Stromal Tumors
- Gastritis, Giant Hypertrophic
- Gastritis, Chronic, Erosive
- Galloway-Mowat Syndrome
- Galactosemia
- Fukuyama Type Congenital Muscular Dystrophy
- Fucosidosis
- Fryns Syndrome
- Fructose Intolerance, Hereditary
- Frontonasal Dysplasia
- Frontofacionasal Dysplasia
- Froelich Syndrome
- Frey Syndrome
- Freeman Sheldon Syndrome
- Fraser Syndrome
- Fragile X Syndrome
- Fox Fordyce Disease
- Fournier Gangrene
- Fountain Syndrome
- Formaldehyde Poisoning
- Glycogen Storage Disease Type III
- Food Protein-Induced Enterocolitis Syndrome
- Focal Dermal Hypoplasia
- Floating Harbor Syndrome
- Fitz Hugh Curtis Syndrome
- Filippi Syndrome
- Filariasis
- Fibrous Dysplasia
- Fibrosing Mediastinitis
- Fibromuscular Dysplasia
- Fibrodysplasia Ossificans Progressiva
- FG Syndrome Type 1
- Fetal Valproate Syndrome
- Fetal Retinoid Syndrome
- Fetal Hydantoin Syndrome
- Fetal Alcohol Syndrome
- Ferroportin Disease
- Femoral Facial Syndrome
- Felty Syndrome
- Fascioliasis
- ASAH1-Related Disorders
- Fanconi Anemia
- Familial Partial Lipodystrophy
- Familial Mediterranean Fever
- Familial Lipoprotein Lipase Deficiency
- Familial Isolated Hypoparathyroidism
- Primary Familial Brain Calcification
- Familial Hypophosphatemia
- Familial Hypercholesterolemia
- Familial Eosinophilic Cellulitis
- Familial Encephalopathy with Neuroserpin Inclusion Bodies
- Familial Cold Autoinflammatory Syndrome
- Familial Adenomatous Polyposis
- Factor XIII Deficiency
- Factor XII Deficiency
- Factor XI Deficiency
- Factor VII Deficiency
- Facioscapulohumeral Muscular Dystrophy
- Fabry Disease
- Biliary Atresia
- Ewing Sarcoma
- Evans Syndrome
- Essential Tremor
- Essential Thrombocythemia
- Essential Iris Atrophy
- Esophageal Atresia and/or Tracheoesophageal Fistula
- Erythropoietic Protoporphyria and X-Linked Protoporphyria
- Erythromelalgia
- Erythema Multiforme
- Erysipelas
- Erdheim Chester Disease
- Epitheliopathy, Acute Posterior Multifocal Placoid Pigment
- Epidermolytic Ichthyosis
- Epidermolysis Bullosa
- Epidermal Nevus Syndromes
- Eosinophilic Gastroenteritis
- Eosinophilic Fasciitis
- Eosinophilic Esophagitis
- Eosinophilia-Myalgia Syndrome
- Enterobiasis
- Endomyocardial Fibrosis
- Infective Endocarditis
- Endocardial Fibroelastosis
- Encephalocele
- Encephalitis, Japanese
- Encephalitis, Herpes Simplex
- Empty Sella Syndrome
- Emphysema, Congenital Lobar
- Emery Dreifuss Muscular Dystrophy
- Ellis Van Creveld Syndrome
- Elephantiasis
- Eisenmenger Syndrome
- Ehlers Danlos Syndromes
- Ectrodactyly Ectodermal Dysplasia Cleft Lip/Palate
- Ectodermal Dysplasias
- Meier-Gorlin Syndrome
- Eales Disease
- Myotonic Dystrophy
- Asphyxiating Thoracic Dystrophy
- Dystonia
- Dysplasia Epiphysealis Hemimelica
- Dyskeratosis Congenita
- Leri-Weill Dyschondrosteosis
- Dysautonomia, Familial
- Dyggve Melchior Clausen syndrome
- Duodenal Atresia or Stenosis
- Duchenne Muscular Dystrophy
- Dubowitz Syndrome
- Dubin Johnson Syndrome
- Duane syndrome
- Dravet Syndrome
- Dracunculosis
- DOOR Syndrome
- Distal Myopathy
- Congenital Sucrase-Isomaltase Deficiency
- Dilatation of the Pulmonary Artery, Idiopathic
- Diffuse Pulmonary Lymphangiomatosis
- Diencephalic Syndrome
- Diastrophic Dysplasia
- Dextrocardia with Situs Inversus
- Desmoid Tumor
- Dermatomyositis
- Dermatitis Herpetiformis
- Dercum's Disease
- Depersonalization Disorder
- Denys-Drash Syndrome
- Dentinogenesis Imperfecta Type III
- Dentin Dysplasia Type II
- Dentin Dysplasia Type I
- Dent Disease
- C3 Glomerulopathy: Dense Deposit Disease and C3 Glomerulonephritis
- Dengue Fever
- Dejerine-Sottas Syndrome
- Degos Disease
- De Santis Cacchione Syndrome
- De Barsy Syndrome
- Danon Disease
- Dandy Walker Malformation
- Cytomegalovirus Infection
- Cytochrome C Oxidase Deficiency
- Cystinuria
- Cystinosis
- Cysticercosis
- Cystic Fibrosis
- Cyclic Vomiting Syndrome
- Cyclic Neutropenia
- Cutis Marmorata Telangiectatica Congenita
- Cutis Laxa
- Cutaneous T-Cell Lymphomas
- Cushing Syndrome
- Cryptococcosis
- Crouzon Syndrome
- Cronkhite-Canada Syndrome
- Crigler Najjar Syndrome
- Cri du Chat Syndrome
- Creutzfeldt Jakob Disease
- Craniometaphyseal Dysplasia
- Craniofrontonasal Dysplasia
- Costello Syndrome
- Corticobasal Degeneration
- Cornelia de Lange Syndrome
- Corneal Dystrophies
- Cor Triatriatum
- Functional Neurological Disorder
- Conradi Hünermann Syndrome
- Congenital Plasminogen Deficiency
- Congenital Varicella Syndrome
- Congenital Pulmonary Lymphangiectasia
- Congenital Muscular Dystrophy
- Congenital Lactic Acidosis
- Congenital Hyperinsulinism
- Congenital Generalized Lipodystrophy
- Congenital Fibrosis of the Extraocular Muscles
- Congenital Fiber Type Disproportion
- Congenital Disorders of Glycosylation
- Congenital Central Hypoventilation Syndrome
- Congenital Bilateral Perisylvian Syndrome
- Congenital Adrenal Hyperplasia
- Cone Dystrophy
- Common Variable Immune Deficiency
- Colorado Tick Fever
- Collagen Type VI-Related Disorders
- Cohen Syndrome
- Cogan Reese Syndrome
- Coffin Siris Syndrome
- Coffin Lowry Syndrome
- Cockayne Syndrome
- Coats Disease
- Cluster Headache
- CLOVES Syndrome
- Cleidocranial Dysplasia
- Classic Hereditary Hemochromatosis
- Citrullinemia Type 1
- Ciguatera Fish Poisoning
- Cicatricial Alopecia
- Churg Strauss Syndrome
- Chronic Myelogenous Leukemia
- Chronic Lymphocytic Leukemia
- Chronic Intestinal Pseudo-Obstruction
- Chronic Inflammatory Demyelinating Polyneuropathy
- Chronic Granulomatous Disease
- Chronic Eosinophilic Pneumonia
- Mosaic Trisomy 9
- Trisomy 9p (Multiple Variants)
- Chromosome 9, Tetrasomy 9p
- Chromosome 9, Partial Monosomy 9p
- Chromosome 9 Ring
- Chromosome 8, Monosomy 8p
- Chromosome 7, Partial Monosomy 7p
- Chromosome 6, Partial Trisomy 6q
- Chromosome 6 Ring
- Chromosome 5, Trisomy 5p
- Chromosome 4, Trisomy 4p
- Chromosome 4, Partial Trisomy Distal 4q
- Chromosome 4, Monosomy Distal 4q
- Chromosome 4q Deletion
- Chromosome 3, Trisomy 3q2
- Chromosome 3, Monosomy 3p
- Chromosome 22q11.2 Deletion Syndrome
- Mosaic Trisomy 22
- Chromosome 22 Ring
- Chromosome 21 Ring
- Chromosome 18q- Syndrome
- Chromosome 18, Tetrasomy 18p
- Chromosome 18, Monosomy 18p
- Chromosome 18 Ring
- Chromosome 15, Distal Trisomy 15q
- Chromosome 15 Ring
- Chromosome 14, Trisomy Mosaic
- Chromosome 14 Ring
- Chromosome 13, Partial Monosomy 13q
- Chromosome 11, Partial Trisomy 11q
- Chromosome 11, Partial Monosomy 11q
- Chromosome 10, Monosomy 10p
- Chromosome 10, Distal Trisomy 10q
- Choroiditis, Serpiginous
- Choroideremia
- Chordoma
- Chondrocalcinosis 2
- Cholesteryl Ester Storage Disease
- Cholera
- Acute Cholecystitis
- Cholangiocarcinoma
- Chilaiditi's Syndrome
- Chikungunya
- Chiari Malformations
- Chiari Frommel Syndrome
- Chediak Higashi Syndrome
- CHARGE Syndrome
- Charcot-Marie-Tooth Disease
- Chandler's Syndrome
- Cervical Teratoma
- Cervical Dystonia
- Cerebrotendinous Xanthomatosis
- Cerebrocostomandibular Syndrome
- Cerebro Oculo Facio Skeletal Syndrome
- Cerebral Palsy
- Cerebellar Degeneration, Subacute
- Cerebellar Agenesis
- Centronuclear Myopathy
- Central Pain Syndrome
- Central Diabetes Insipidus
- Central Core Disease
- CDKL5 Deficiency Disorder
- Cavernous Malformation
- Caudal Regression Syndrome
- Catel Manzke Syndrome
- Catamenial Pneumothorax
- Cat Eye Syndrome
- Castleman Disease
- Carpenter Syndrome
- Caroli Disease
- Carnosinemia
- Carnitine Palmitoyltransferase 1A Deficiency
- Systemic Primary Carnitine Deficiency
- Carney Complex
- Cardiofaciocutaneous Syndrome
- Carcinoid Syndrome
- Carbamoyl Phosphate Synthetase I Deficiency
- CARASIL
- Candidiasis
- Canavan Disease
- Camurati-Engelmann Disease
- Campomelic Syndrome
- CADASIL
- C Syndrome
- Bullous Pemphigoid
- Buerger's Disease
- Budd Chiari Syndrome
- Brugada Syndrome
- Brucellosis
- Brown Syndrome
- Brown Séquard Syndrome
- Bronchopulmonary Dysplasia
- Bronchiolitis Obliterans Organizing Pneumonia
- Branchiootorenal Spectrum Disorders
- Branchio Oculo Facial Syndrome
- Bowenoid Papulosis
- Bowen Hutterite Syndrome
- Bowen Disease
- Botulism
- Börjeson-Forssman-Lehman Syndrome
- Blue Rubber Bleb Nevus syndrome
- Blue Diaper Syndrome
- Bloom Syndrome
- Blepharophimosis, Ptosis, Epicanthus Inversus Syndrome
- Blastomycosis
- Bladder Exstrophy-Epispadias-Cloacal Exstrophy Complex
- Björnstad Syndrome
- Birt-Hogg-Dubé Syndrome
- Biotinidase Deficiency
- Binswanger Disease
- Binder Type Nasomaxillary Dysplasia
- Bile Acid Synthesis Disorders
- Best Vitelliform Macular Dystrophy
- Berylliosis
- Bernard-Soulier Syndrome
- Benign Paroxysmal Positional Vertigo
- Benign Essential Blepharospasm
- Bell's Palsy
- Bejel
- Behçet's Syndrome
- Beckwith-Wiedemann Syndrome
- Congenital Contractural Arachnodactyly
- Juvenile CLN3 Disease
- Bartter Syndrome
- Bartonellosis
- Barth Syndrome
- Bardet-Biedl Syndrome
- Barakat Syndrome
- Banti's Syndrome
- Balo Disease
- Baller-Gerold Syndrome
- Balantidiasis
- Babesiosis
- Autosomal Recessive Polycystic Kidney Disease
- Autosomal Recessive Hyper IgE Syndrome
- Autosomal Dominant Porencephaly Type I
- Autosomal Dominant Polycystic Kidney Disease
- Autosomal Dominant Tubulo-Interstitial Kidney Disease
- Autosomal Dominant Hyper IgE Syndrome
- Autosomal Dominant Hereditary Ataxia
- Autoimmune Polyendocrine Syndrome Type II
- Autoimmune Blistering Diseases
- Atypical Hemolytic Uremic Syndrome
- Atrioventricular Septal Defect
- Atrial Septal Defects
- Atransferrinemia
- Friedreich's Ataxia
- Ataxia with Vitamin E Deficiency
- Ataxia Telangiectasia
- Astrocytoma
- Aspergillosis
- Aspartylglycosaminuria
- Asherson's Syndrome
- Asherman's Syndrome
- Arthrogryposis Multiplex Congenita
- Arthritis, Infectious
- Arteritis, Takayasu
- Giant Cell Arteritis
- Arteriovenous Malformation
- Arterial Tortuosity Syndrome
- Argininosuccinic Aciduria
- Arginase-1 Deficiency
- Arachnoid Cysts
- Autoimmune Polyglandular Syndrome Type 1
- Apraxia
- Apnea, Infantile
- Aplasia Cutis Congenita
- Apert Syndrome
- Antley-Bixler Syndrome
- Antithrombin Deficiency
- Antiphospholipid Syndrome
- Anthrax
- Tooth Agenesis
- Aniridia Cerebellar Ataxia Mental Deficiency
- Aniridia
- Angioimmunoblastic T-Cell Lymphoma
- Hereditary Angioedema
- Angelman Syndrome
- Anencephaly
- Anemias, Sideroblastic
- Anemia, Pernicious
- Anemia, Megaloblastic
- Hereditary Spherocytosis
- Hereditary Nonspherocytic Hemolytic Anemia
- Cold Agglutinin Disease
- Anemia, Hemolytic, Acquired Autoimmune
- Diamond Blackfan Anemia
- Anemia of Chronic Disease
- Partial Androgen Insensitivity Syndrome
- Andersen Disease (GSD IV)
- Anaplastic Astrocytoma
- Amyotrophic Lateral Sclerosis
- Amyloidosis
- Amniotic Band Syndrome
- Amelogenesis Imperfecta
- Ameloblastoma
- Ameloblastic Carcinoma
- Alveolitis, Extrinsic Allergic
- Alveolar Soft Part Sarcoma
- Alveolar Capillary Dysplasia with Misalignment of Pulmonary Veins
- Alternating Hemiplegia of Childhood
- Alström Syndrome
- Alport Syndrome
- Alpha-Mannosidosis
- Alpha-1 Antitrypsin Deficiency
- Alpha Thalassemia X-linked Intellectual Disability Syndrome
- Alpers Disease
- Alopecia Areata
- Alkaptonuria
- Alexander Disease
- Alagille Syndrome
- ALAD Porphyria
- AIDS Dysmorphic Syndrome
- Aicardi Syndrome
- Ahumada-Del Castillo Syndrome
- Agranulocytosis, Acquired
- Agenesis of Corpus Callosum
- Agammaglobulinemia
- African Iron Overload
- Congenital Afibrinogenemia
- AEC Syndrome
- Adult Onset Still's Disease
- X-Linked Adrenoleukodystrophy
- Adie Syndrome
- Adenylosuccinate Lyase Deficiency
- Adenoid Cystic Carcinoma
- Addison's Disease
- Adams Oliver Syndrome
- Acute Respiratory Distress Syndrome
- Acute Intermittent Porphyria
- Acute Eosinophilic Pneumonia
- Acute Disseminated Encephalomyelitis
- ACTH Deficiency
- Acromicric Dysplasia
- Acromesomelic Dysplasia
- Acromegaly
- Acrodysostosis
- Acrodermatitis Enteropathica
- Acrocallosal Syndrome, Schinzel Type
- Acquired Neuromyotonia
- Acquired Lipodystrophy
- Acquired Hemophilia
- Acquired Aplastic Anemia
- Acoustic Neuroma
- Propionic Acidemia
- Acidemia, Methylmalonic
- Isovaleric Acidemia
- Achondroplasia
- Achondrogenesis
- Achard Thiers Syndrome
- Achalasia
- Aceruloplasminemia
- Acanthosis Nigricans
- Acanthocheilonemiasis
- Ablepharon-Macrostomia Syndrome
- Abetalipoproteinemia
- Aarskog Syndrome
- Glutathione Synthetase Deficiency
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- Illumina
- Kiniksa
- Optum Frontier Therapies
- Vanda Pharmaceuticals Inc.
- Sigilon Therapeutics
- Taysha Gene Therapies
- Viela Bio
- Ascendis Pharma
- Neurogene
- Acadia Pharmaceuticals
- Harmony Biosciences, LLC
- X4 Pharmaceuticals
- Shape Therapeutics
- Orna Therapeutics, Inc
- Crisprtx
- Dante Labs
- Deciphera
- AstraZeneca
- Helixmith
- Parexel
- Momenta Pharmaceuticals
- Applied Therapeutics
- Foundation Medicine
- Medidata
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