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Posts by category
- Category: Advocacy
- NORD Issues New Rare Disease DayⓇ Rallying Cry: Show Your Stripes™!
- Rare Disease Advocates Share Ideas for How to Mark Rare Disease Day 2019
- NORD Issues Statement on FDA’s Impending Expanded Access Call Center
- Rare Disease Day 2019 is Only 10 Weeks Away!
- Orphan Drug Act Resolution Introduced in Congress
- Featured Student: Jennifer Shoskes
- Patient Advocates Warn Against New Insurance & PBM Policy That Increases Patient Out-of-Pocket Drug Costs
- Three Big Reasons Not to Miss the 2018 NORD Rare Summit
- 2018 NORD Rare Summit to Feature Distinguished Speakers Discussing a New Era of Patient-Focused Innovation
- NORD Invites Video Submissions on Advocacy Experience
- NORD Responds to the Administration’s “Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs”
- FDA Commissioner Dr. Scott Gottlieb Confirmed as 2018 NORD Rare Summit Keynote Speaker
- NORD Issues Statement on Recent CMS Actions Pertaining to Patient Access Within Medicaid Formularies
- 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 Launches RareInsights, New Initiative to Support Data-Driven Advocacy, and Presents 5 Myths About Orphan Drugs and the Orphan Drug Act
- NORD Issues Statement Regarding House Passage of the Right to Try Act (S.204)
- NORD 35th Anniversary Blog Series: The Early Years (1980s)
- 35 Ways and Growing: NORD’s Service to the Rare Disease Community
- Estelle Benson, Founder of GBS|CIDP Foundation International: Abbey S. Meyers 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
- TAKE ACTION: Join NORD & Over 100 Patient Organizations in Supporting the RARE Act
- 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 Rare Action Network® 2018 State Report Card
- 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
- NORD Sends Letter to House of Representatives on Right to Try Act
- NORD Issues Statement on Confirmation of New Secretary of HHS, Alex Azar
- NORD Launches 7,000 Mile Rare Movement
- Your Help is Needed to #SaveCHIP
- 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
- Join Us for Rare Disease Day®
- NORD Issues Statement in Response to Congressional Consideration of the Tax Cuts and Jobs Act Conference Report
- 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
- Rare Disease Advocates Host a Save the Orphan Drug Tax Credit Rally
- 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 Issues Statement in Response to Senate Finance Committee’s Proposal to Weaken the Orphan Drug Tax Credit
- More Than 200 Patient Groups Call On Congress to Reinstate the Orphan Drug Tax Credit in the Tax Cuts and Jobs Act
- NORD Issues Statement in Response to Proposed Repeal of the Orphan Drug Tax Credit (ODTC)
- Join NORD & SSA in Forum on Compassionate Allowances & Rare Diseases
- NORD Releases New Documentary called “Good Morning Peyton”
- NORD Issues Statement on Senate Leadership’s Recent Update on “Graham-Cassidy” Proposal
- Protecting Your Healthcare Coverage
- NORD Issues Statement Opposing the “Graham-Cassidy” ACA Replacement Plan
- Over 140 Patient Organizations Support the Orphan Drug Tax Credit
- 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 Senate passage of the Food and Drug Administration Reauthorization Act of 2017
- NORD Issues Statement on the Latest ACA Repeal and Replace Developments
- The BCRA and Healthcare Reform: NORD’s Latest Update
- 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 Rare Action Network℠ Releases Summer 2017 State Policy Legislative Tracker
- Rare Disease Advisory Council Bill Moves through Pennsylvania House
- 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 Issues Statement on the Better Care Reconciliation Act of 2017
- NORD Leads Rare Disease Community Days of Action Campaign to Protect Healthcare Coverage
- NORD Issues Statement on Confirmation of New FDA Commissioner, Dr. Scott Gottlieb
- NORD Issues Statement on Today’s Passage of the American Health Care Act
- 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
- Advocacy Alert: Tell Congress to Act Now and Pass FDA User Fee Agreements
- NORD Rare Action Network℠ Releases Spring 2017 State Policy Legislative Tracker
- 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
- Cynthia Tifft, M.D., Ph.D – 2017 Rare Impact Award Honoree
- 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
- Brandon Hudgins – 2017 Rare Impact Award Honoree
- NORD Issues Statement Opposing the Preserving Employee Wellness Programs Act (H.R.1313)
- 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
- NORD Issues Statement on President Trump’s Address to Congress
- Orphan Drugs Represent 41 Percent of All New Medications
- Reflections on FDA Listening Session
- President Signs 21st Century Cures Medical Innovation Bill Into Law
- State Report Card to Help People with Rare Diseases
- Expansion of Medical Foods Coverage for Military Families Heads to the President, Having Passed in the Senate
- Senate Passes Landmark 21st Century Cures Act
- 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
- NORD Ready to Address New Challenges Based on Election Results
- $1.1 Billion in Federal Funding to Fight the Zika Virus
- 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)
- 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 Publishes Expanded Access FAQ
- NORD Issues Statement in Response to PDUFA VI Commitment Letter
- Advocacy Alert: Illinois Rare Disease Commission Bill
- 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
- NORD Applauds Senate for Authorizing Medical Foods Coverage for the Military in National Defense Authorization Act (S.2943)
- Today, Tell Congress We Need #CuresNow
- RareAction Network℠ Releases May/June State Policy Legislative Tracker
- NORD Summit 2016
- NORD to Hold Rare Diseases Roundtable
- New Paper Addresses Health Care Costs and Patient Assistance Programs
- RareAction Network℠ Releases April State Legislative Tracker
- Advocacy Alert: Illinois Edges Closer to Establishing Rare Disease Commission
- NORD Founder Publishes Memoir Detailing Her Tireless Work as Parent-Advocate
- Today is Rare Disease Day®
- NORD Issues Statement on New FDA Commissioner Robert Califf, M.D.
- Rare Disease Day Video for 2016 Launched
- Wall Street Journal Publishes NORD Letter to the Editor
- FDA Approves 21 Orphan Drugs for Rare Diseases in 2015, Sets Record for Second Consecutive Year
- Help Tara Pass School Bus Legislation
- What the Omnibus Bill Means for Rare Diseases
- Get Involved in Your State on Rare Disease Day
- Be a Hero: Help NORD Empower the Rare Disease Community in Washington D.C. and in all 50 States.
- 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 Releases First-Ever State Progress Report for Rare Diseases
- Raise Your Voice: Rare Disease Day 2016 is Coming
- NORD Issues Statement Applauding the Approval of Ensuring Access to Clinical Trials Act
- American Plasma Users Coalition (A-PLUS) Applauds Recent Move by Department of Health and Human Services to Limit Out-of-Pocket Costs
- NORD Issues Statement on FDA Rejection of Citizen Petition
- Rare Disease Day 2016 Theme and Slogan Announced!
- 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
- Rare Action™ Road Tour, Salt Lake City, UT
- Rare Action™ Road Tour, Salt Lake City, UT
- NORD Signs Letter with Center for Responsible Science Calling for Changes at the FDA
- Rare Action™ Road Tour, Denver, CO
- Taking Rare Action™ on the Road
- NORD Files Amicus Brief to Protect Drug Development Incentives in the Orphan Drug Act
- 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
- Rare Action™ Road Tour, Atlanta, GA
- 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
- Impact of the Orphan Drug Tax Credit on Treatments for Rare Diseases
- NORD Submits Final Comments on 21st Century Cures Act
- Global Voice for Rare Disease Patients Launches Today: Rare Diseases International
- NORD Issues Statement on Today’s Approval of the 21st Century Cures Initiative
- FDA Public Meeting on Chagas Disease Patient-Focused Drug Development
- The Will to Live and the Strength for a Cure
- Saving Eliza
- Multiple Endocrine Neoplasia Type 1: One Family’s Fight
- Taking Action for ACD
- The Will to Live and the Strength for a Cure
- 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
- NORD at Capitol Hill Hearing: Patients Have Unique Perspective That Needs to be Heard
- Knowing More About Noonan
- NORD Recommendations for Advancing Drug Discovery, Development, and Delivery
- 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
- 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”
- Our Shared Vision for Rare Disease Day
- Building on Policy Momentum
- 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
- 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
- Campaign to Educate State Legislators for Rare Disease Day
- Patients Will Benefit From New Public-Private Partnership
- Proposed Rules for Health Insurance Market Reforms and Essential Health Benefits
- Category: Events
- The 2019 Rare Impact Awards will take off at Space Center Houston, Saturday, June 22!
- Chat, Chew, and Be Challenged! Lunchtime Learning at the NORD Rare Summit
- Patient/Caregiver Opening Address Announced for NORD Rare Summit
- Join us at the 2018 NORD Rare Summit
- A Celebration of NORD’s 35th Anniversary
- Save the Date for NORD’s 2018 Rare Summit
- Watch LIVE: NORD’s 35th Anniversary & Rare Impact Awards Celebration
- Robert Campbell, Jr., M.D.: 2018 Lifetime Achievement Award Honoree
- Early Diagnosis: Perspectives from NORD Student Chapter Leader, Harjot Randhawa
- Nominations for the Rare Impact Awards Close Friday, January 12
- Registration Open!
- FDA Announces First Gene Therapy in U.S.
- NORD Announces Keynote Speakers for 2017 Rare Diseases and Orphan Products Breakthrough Summit
- NORD Announces Honorees for the 2017 Rare Impact Awards
- Musical Act Announced for Rare Impact Awards on May 18th
- Rare Disease Summer Family Camp: A Place to Call Home
- Join NORD and ABC News for a Rare Disease Day® Tweetchat on Feb. 28
- FDA Commissioner Dr. Robert Califf Delivers Keynote Address at NORD Summit
- Nominations Open for NORD’s 2017 Rare Impact Awards
- Rare Diseases & Orphan Products Breakthrough Summit Speakers
- Running for Rare Named an Official Charity Partner
- Key Dates Approaching for NORD Summit
- FDA Commissioner Dr. Robert Califf to Speak at NORD Summit in October
- See What’s New and Exciting at the NORD 2016 Summit, Oct. 17-18!
- Join the Team: Running for Rare Takes on Hartford
- Register for a FREE Webinar on the Undiagnosed Diseases Network (UDN)
- Introducing the 2016 Rare Disease Day® Highlights Video
- 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
- Apply Now: The Hole in the Wall Gang Camp Hosts First-Ever Rare Disease Family Camp
- Join the Running for Rare Team!
- Register for the 2016 Rare Impact Awards
- Rare Disease Day 2016 Theme and Slogan Announced
- Rare Disease Artists’ Work Traveling Globe to Raise Awareness
- NORD Webinar Series
- Rare Disease Day
- Category: Featured News
- 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
- NORD Expands and Enhances its Rare Disease Database®, a Primary Resource for Millions of People Affected by Rare Diseases
- New Study Finds Orphan Drug Exclusivity Working As Intended, On-Market Orphan Drug Prices Rise Slower than Common Drugs
- Year-End Message from NORD’s President and CEO
- 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 Creates Rare Disease Patient & Caregiver Resource Center
- 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
- NORD 35th Anniversary Blog Series: 2010-2018 (Present)
- Healthline and NORD Announce Recipients of 2018 Stronger Scholarships
- NORD Issues Statement on the Passing of Dr. Robert M. Campbell
- NORD’s RareEDU™ Launches Video Addressing a Topic Vital to Today’s Rare Disease Community, Gene Therapy: Your Questions Answered
- Moving documentary on Joshua Frase Foundation, Nibs – the matriarch of the first ever MTM dog colony, and the power of patient advocacy
- 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
- New Research Funding Opportunities Available from NORD
- NORD statement in response to American Patients First, the Trump Administration Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs
- Richard Pazdur, M.D.: 2018 Public Health Leadership Award Honoree
- Chris Ulmer: 2018 Rare Impact Award Honoree
- Neurology Reviews® and NORD Publish the 2018 Rare Neurological Disease Special Report
- Changing the Landscape of Rare Disease Research
- NORD to Collaborate with FDA on Pilot Patient Engagement Activity
- Current Funding Opportunity: NORD Research Grants for Rare Diseases
- NORD Awards Five New Grants for Rare Disease Research
- Together, we can be stronger for those that need us.
- NORD Publishes Information for Physicians and Patients on Three Rare Diseases
- 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 Publishes New Rare Disease Report on Shprintzen Goldberg Syndrome (SGS)
- Downloadable Hurricane Harvey Emergency Relief Resources
- NORD Publishes New Report on Sitosterolemia
- NORD Publishes New Rare Disease Report on Retinal Vasculopathy with Cerebral Leukodystrophy (RVCL)
- NORD Publishes New Rare Disease Report on Bosma arhinia microphthalmia (BAM) syndrome
- NORD Releases Statement on Orphan Drug Assessment & Pricing Summit
- NORD Publishes Report on Hereditary Sensory Autonomic Neuropathy Type 1E
- Sign-on letter from 120 patient organizations urging Congress to reauthorize FDA User Fee Agreements
- Christine Grube – 2017 Rare Impact Award Honoree (Awarded Posthumously)
- Dr. Robert J. Desnick, MD, Ph.D – 2017 Rare Impact Award Honoree
- NORD Unveils “Do Your Share for Rare,” Year-long Campaign for Rare Disease Public Awareness
- Raise Your Hand for Rare Disease Research
- Family Moves Across State Lines to Receive Better Access
- A Message from Our President and CEO
- A New Partnership that Helps Raise Awareness of Rare Diseases
- Happy Father’s Day from NORD
- NORD Appoints Genetics and Metabolic Specialist Marshall L. Summar M.D. as Chairman of the Board
- NORD Speakers Bureau
- NORD Members Share Great Resources for ER Visits During #RareERChat
- Watch Live: Tomorrow’s Rare Impact Awards Ceremony
- Desiree Lyon Howe: 2016 Rare Impact Award Honoree
- NORD Website Reaches 1 Million Monthly Visits
- Dr. Stephen Cederbaum: 2016 Rare Impact Award Honoree
- Noah Victoria: 2016 Rare Impact Award Honoree
- NORD and Trio Health to Provide Real-World Insights to Improve Access and Quality of Care for More Than 7,000 Rare Diseases
- A Special Thank You to Volunteers in the Rare Disease Community
- Neurology Reviews Publishes The Rare Neurological Diseaes Special Report
- Running on Air for Rare Disease Day
- NORD Establishes Rare Disease Patient/Caregiver Speakers Bureau
- FDA Invites Patient Organizations to Organize Their Own Meetings on the PFDD Model
- Cure HHT Foundation
- CCHS Family Network
- 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
- Rett Syndrome Research Trust
- Liam Cruz’ Diagnostic Journey
- Target Cancer Foundation
- Narcolepsy Network
- The Undiagnosed: Orphans of the Rare Disease Community
- Dravet Syndrome Foundation
- Jill Zeigler’s Story: Life with aHUS
- HLRCC Family Alliance
- 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 Disease Report and NORD Announce Editorial Collaboration
- Member Spotlight: CLOVES Syndrome Community
- Member Spotlight: LGDA
- 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
- Life is a Special Occasion
- NORD Celebration Highlights the Courage of Children and Adults Living with Rare Diseases
- Portraits of Courage Celebration
- NORD Launches Rare Action Network
- The Will to Live and the Strength for a Cure
- Are Civility and Mature Behavior Really Too Much to Expect?
- FDA Creates New Web-Based Educational Tool About the Agency
- BioNJ Presents Patient Advocate Award to NORD
- Statement on Congressman Waxman’s Retirement
- Even “Well Known” Rare Diseases Pose Diagnosis Problems
- 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: Get Involved
- 35 Ways to Celebrate the 35th Anniversary of NORD
- Penn State Graduate Rare Disorder Survey
- Celebrating Awareness: One-Year Anniversary of Rare Disease of the Day
- Becoming a Marathon Runner
- NORD Hosts Tweetchat with Texas Children’s Hospital on Navigating the Pediatric ER (May 26)
- Join NORD and ABC News for a Rare Disease Day Tweetchat on March 1
- Category: Industry
- Category: Medical
- NORD Publishes Report on Post-Transplant Lymphoproliferative Disease
- NORD Seeks Proposals for CME Partnership
- NORD Publishes New Report on RYR-1 Related Diseases
- NORD Publishes New Rare Disease Report on CARD9 Deficiency
- NORD Publishes Physician Guide to Cutaneous T-Cell Lymphoma
- NORD Publishes Physician Guide to Mitochondrial Myopathies
- 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
- New Video from NORD Promotes Awareness of Rare Movement Disorder Neurogenic Orthostatic Hypotension
- Thoughtful Biosimilars Policy Is Best Way to Ensure Optimum Patient Outcomes
- 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
- Creative Collaborations May Be Key to Reducing Chemotherapy Risks
- Category: Patient Stories
- Giving Spotlight: Honoring Mom-Mom’s Legacy with Research and Awareness
- Mackenzie’s Mission
- A Group No One Applies to Join
- 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
- Zan’s Journey: Diagnosed with SMS
- Category: Patients & Members
- 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
- International Pemphigus & Pemphigoid Foundation Launches Largest-Ever Study of Pemphigus and Pemphigoid
- NORD Publishes Report on Spontaneous Intracranial Hypotension
- First Natural History Registry for Necrotizing Enterocolitis
- Largest-Ever Study of SYNGAP1 (MRD5), Linked to Autism, was Launched
- Fibrous Dysplasia Foundation and NORD Launch Cutting-Edge Study of Fibrous Dysplasia/McCune-Albright Syndrome
- September is Children’s Cardiomyopathy Awareness Month
- Guest Blog: Tour For A Cure
- Happy Mother’s Day from NORD!
- Notification (2/25/2016): Patient Assistance Programs
- 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
- 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
- Barretstown – a Serious Fun Camp!
- Category: Press Releases
- Updated Study Analyzes Use and Cost of Orphan Drugs
- NORD and the Orphan Drug Act Celebrate 35th Anniversaries
- New Study Examines Use and Cost of Orphan Drugs
- NORD Announces Three New Hires to Lead Research, Education and Business Development Programs
- NORD Names New Director of Membership
- NORD and Neurology Reviews Publish Special Report
- NORD Awards New Research Grants for Rare Disease Research
- NORD Announces 20 Rare Disease Patient Groups Selected to Develop Natural History Studies as Part of FDA Cooperative Agreement
- NORD Developing 20 Natural History Studies for 20 Rare Diseases
- NORD Issues Statement on NIH Appointment of New Director for Office of Rare Diseases Research
- Category: Rare Disease Day
- Category: Research
- Category: Sticky Posts for Advocate
- Category: Sticky Posts for Clinicians and Researchers
- Category: Sticky Posts for Homepage
- Category: Sticky Posts for Industry
- Category: Sticky Posts for Patient Organizations
- Category: Sticky Posts for Patients and Families
- Category: Uncategorized
- 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
- 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
Venues
- 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
- 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
- 8th Biennial Conference on Glut1 Deficiency
- Living Rare, Living Stronger NORD Patient & Family Forum
- 19th Edition on World Pathology Week
- AAIDA 2nd Annual Taking the Mask off Immune Disorders
- AIU 2019 Conference
- 28th International Conference on Insights in Ophthalmology
- 15th International Conference on Surgical Pathology and Cancer Diagnosis
- 8th Edition of International Conference on Internal Medicine & Patient Care
- 22nd International Conference on New Horizons in Cardiology & Cardiologists Education
- Swing for The Kids 2019
- Rare Disease Day @ Iowa State
- N4R Ham Radio Rare Disease Day Special Event
- World Rare Disease Day Rally of Charlotte
- Rare Disease Family Night
- Moebius Syndrome Awareness Day
Organizations
- 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
- COL4A1 Foundation
- Bili Project Foundation
- ACDY5.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
- 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
- Lipoprotein A Foundation
- 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
- The Transverse Myelitis Association
- Autoimmune Encephalitis Alliance
- Spinal CSF Leak Foundation
- National Health Council
- Charlotte & Gwenyth Gray Foundation to Cure Batten Disease
- Mitochondrial Medicine Society
- NGLY1.org
- The Myelin Project
- International Society for Cutaneous Lymphomas
- STXBP-1 Fund
- 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
- ISMRD
- Child Growth Foundation
- Rett Syndrome Research Trust
- The JMML Foundation
- CMTC-OVM US
- 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 Research Society, Inc.
- Ocular Melanoma Foundation
- Jared's Juggernaut, Inc.
- Julia's Wings Foundation, Inc.
- Community United for Research and Education of Ocular Melanoma
- Morgan Leary Vaughan Fund, Inc.
- 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
- Council for Bile Acid Deficiency Diseases
- Klippel-Feil Syndrome Alliance
- PANDAS Network.org
- ALD Life
- 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
- Potentials Foundation
- 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 VIP Connect
- 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
- Fibrous Dysplasia Foundation
- 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)
- MDJunction Online Support Group
- 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
- 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
- National Organization of Vascular Anomalies
- 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 E-Support Site
- American Partnership for Eosinophilic Disorders (APFED)
- Jain Foundation Inc.
- The Association For X and Y Chromosome Variations
- 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.
- Colorectal Cancer Network
- 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
- CLIMB 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.
- Basal Cell Carcinoma Nevus Syndrome Life Support Network
- MdDS Balance Disorder Foundation
- Fetal Alcohol Spectrum Disorders Center for Excellence
- XXYY Project
- Bowel Group for Kids Inc.
- GOLD, Global Organisation For Lysosomal Diseases
- 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 Research Foundation
- Sarcoma Alliance
- National Shingles Foundation
- Foundation for Children with Atypical HUS
- 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.
- Childhood Apraxia of Speech Association of North America
- 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
- 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)
- Neuropathy Trust
- United Ostomy Association of Canada
- National Association of Laryngectomee Clubs (UK)
- Diabetes New Zealand
- Mental Health Foundation (UK)
- Prostate Cancer UK
- Lymphoma Association (UK)
- Canadian Hearing Society
- Ovarian Cancer National Alliance
- M.A.G.N.U.M. Migraine Awareness Group: A National Understanding for Migraineurs
- Lymphoma Foundation 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
- Nebraska Advocacy Services, Inc.
- 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)
- Hereditary Multiple Exostoses Support Group
- New Zealand Dystonia Patient Network Inc.
- Vermont Legal Aid
- Mental Health America of Illinois
- National Fetal AntiConvulsant Syndrome Association
- 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
- Neuropathy Association
- 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
- Sarcoid Networking Association
- 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 Syndrome Support Group
- 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.
- American Pain Society
- 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 Registry
- 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
- Incontinentia Pigmenti International Foundation
- Mountain States Genetics Foundation
- National Hospice and Palliative Care Organization
- National Hansen's Disease Programs
- 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
- Narcolepsy Institute
- 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)
- ELA - European Association Against Leukodystrophies
- 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
- Transverse Myelitis Association
- 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
- National Vitiligo Foundation
- 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
- National Congenital CMV Disease 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
- FSH Society, Inc.
- 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
- Lipodystrophy United
- 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
- Christopher S. Burton Syringomyelia Foundation, Inc.
- 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
- 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 Antibody 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 Syndrome
- 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
- Tourette 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 Dysmorphia Syndrome
- Simple Pulmonary Eosinophilia
- Simian B Virus Infection
- Sickle Cell Disease
- Sialidosis
- Sialadenitis
- Shwachman Diamond Syndrome
- SHORT Syndrome
- Short Chain Acyl CoA Dehydrogenase Deficiency (SCAD)
- 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
- 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
- Rickets, Vitamin D Deficiency
- 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
- Reflex Sympathetic Dystrophy 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
- Progressive Symmetric Erythrokeratodermia
- 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
- 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
- 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
- New-Onset Refractory Status Epilepticus (NORSE) and Febrile Infection-Related Epilepsy Syndrome (FIRES)
- 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
- Myopathy, Congenital, Batten Turner Type
- Myhre Syndrome
- Myelodysplastic Syndromes
- Mycosis Fungoides
- Myasthenia Gravis
- Myalgic Encephalomyelitis
- 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
- Macular Degeneration
- 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
- 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
- Idiopathic Neonatal Hepatitis
- Idiopathic Intracranial Hypertension
- Ichthyosis, X Linked
- Ichthyosis, Trichothiodystrophy
- Sjögren-Larsson Syndrome
- Ichthyosis, Netherton Syndrome
- Ichthyosis, Lamellar
- Ichthyosis, Keratosis Follicularis Spinulosa Decalvans
- Ichthyosis, Harlequin Type
- Ichthyosis, Erythrokeratolysis Hiemalis
- Ichthyosis, Erythrokeratodermia Variabilis
- 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
- 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
- Hers Disease
- 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
- Heart Block, Congenital
- 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 VII
- Glycogen Storage Disease Type V
- Glycogen Storage Disease Type IX
- Glycogen Storage Disease Type I
- Glutaricaciduria II
- Glutaricaciduria I
- Glucose-Galactose Malabsorption
- Glucose-6-Phosphate Dehydrogenase Deficiency
- Glucose Transporter Type 1 Deficiency Syndrome
- Glioblastoma Multiforme
- 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
- Farber's Disease
- 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
- Erythrokeratodermia with Ataxia
- 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
- Endocarditis, Infective
- 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
- Ear, Patella, Short Stature Syndrome
- Eales Disease
- Myotonic Dystrophy
- Asphyxiating Thoracic Dystrophy
- Dystonia
- Dysplasia Epiphysealis Hemimelica
- Dyskeratosis Congenita
- Leri-Weill Dyschondrosteosis
- Dysautonomia, Familial
- Dyggve Melchior Clausen syndrome
- Dupuytren's Contracture
- 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 Disease
- 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 4, Monosomy 4q
- 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
- 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
- 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's 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, Psoriatic
- Arthritis, Juvenile Rheumatoid
- Arthritis, Infectious
- Arteritis, Takayasu
- Giant Cell Arteritis
- Arteriovenous Malformation
- Arterial Tortuosity Syndrome
- Argininosuccinic Aciduria
- Arginase Deficiency
- Arachnoiditis
- 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
- Anemia, Hereditary Spherocytic Hemolytic
- Anemia, Hereditary Nonspherocytic Hemolytic
- Anemia, Hemolytic, Cold Antibody
- Anemia, Hemolytic, Acquired Autoimmune
- Anemia, Blackfan Diamond
- Anemia of Chronic Disease
- Androgen Insensitivity Syndrome, Partial
- 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
- 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
- Acidemia Isovaleric
- Achondroplasia
- Achondrogenesis
- Achard Thiers Syndrome
- Achalasia
- Aceruloplasminemia
- Acanthosis Nigricans
- Acanthocheilonemiasis
- Ablepharon-Macrostomia Syndrome
- Abetalipoproteinemia
- Aarskog Syndrome
- Glutathione Synthetase Deficiency
Patient Assistance Programs
- Alipogene Tiparvovec Phase III GLY-CD-001
- Urea Cycle Disorder (UCD) Medical Foods & Supplements
- Autonomic Dysreflexia Copay Assistance
- Lixelle β2M Apheresis Column
- Alpha-1 Antitrypsin Deficiency Premium and Co-Pay Assistance Program
- Lambert Eaton Myasthenic Syndrome (LEMS)
- Congenital Myasthenic Syndromes Clinical Study
- Duchenne Muscular Dystrophy FG-3019
- Hypophosphatasia (HPP)
- Seizures Associated with LGS
- Chorea
- Duchenne Muscular Dystrophy PTC 124-GD-020e-DMD
- Duchenne Muscular Dystrophy PTC124-GD-020-DMD
- Duchenne Muscular Dystrophy PTC124-GD-016-DMD
- Gaucher Disease
- CTX (Cerebrotendinous Xanthomatosus)
- Macular Edema Associated with Retinal Vein Occlusion
- BUPHENYL® Medication Assistance (sodium phenylbuterate)
- Hunter Syndrome
- ITB® Therapy Assistance Lioresal® Intrathecal (baclofen injection)
- Motor Tic Disorder
- CYSTADANE® Medication Assistance (betaine anhydrous for oral solution)
- Cushing's Disease
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Cryopyrin Associated Periodic Syndromes (CAPS)
- Neurogenic Orthostatic Hypotension (NOH)
- IGF-1 Deficiency
- Morquio A Syndrome
- Short Bowel Syndrome (SBS)
- ISIS 420915-CS2 TTR Rx
- Spinal Muscular Atrophy (CS12 SMNRX)
- Spinal Muscular Atrophy (CS3A SMNRX)
- Phenylketonuria (PKU)
- Therakos Photopheresis Therapy
- Cystic Fibrosis - PTC 124-GD-023-CF
- Cystic Fibrosis - PTC 124-GD-021-CF
- Mucopolysaccharidosis Type VI (Maroteaux-Lamy Syndrome)
- Diamond Blackfan Anemia (DBA)
- Congenital Sucrase-Isomaltase Deficiency (CSID)
- Chronic Non-Infectious Uveitis
- Cervical Dystonia (spasmodic torticollis)
Companies
- OptumRx
- Chiesi USA
- Rallybio
- Acceleron Pharma
- Ionis Pharmaceuticals
- UCB Pharma
- Daiichi Sankyo
- BriovaRx
- Orsini Healthcare
- Bayer
- Dompé
- Genevant
- ThinkGen
- PTC Therapeutics
- Orphazyme
- BridgeBio
- United Therapeutics Corporation
- Elysium Health
- Savara, Inc.
- Regeneron
- AVROBIO
- Enzyvant
- Aeglea BioTherapeutics
- LEO Pharma
- Albireo
- Syneos Health
- Sangamo
- ICON
- Harmony Biosciences, LLC
- Moderna Therapeutics
- Zealand Pharma
- Agilis Biotherapeutics Inc
- AveXis, Inc.
- Rhythm Pharmaceuticals
- PhRMA
- Astellas
- Chemistry Rx
- Children's National Health System
- Syros Pharmaceuticals
- Global Blood Therapeutics, Inc.
- Rigel Pharmaceuticals
- Regeneron
- Prevention Genetics
- Grunenthal USA
- Teva Pharmaceuticals
- CTD Holdings, Inc.
- Rocket Pharmaceuticals Ltd.
- Eiger BioPharmaceuticals
- Editas Medicine
- Amgen
- Ovid Therapeautics
- MeiraGTx
- Lysogene
- Zogenix, Inc.
- Orchard Therapeutics Ltd.
- Bristol-Myers-Squibb
- bluebirdbio
- REGENXBIO
- AGTC
- ProMetic Life Sciences
- Akcea Therapeutics
- Hansa Medical
- Alnylam
- Spark Therapeutics
- Chiasma Pharma
- Blueprint Medicines
- Achillion
- Mallinckrodt Pharmaceuticals
- Audentes Therapeutics
- Boehringer-Ingelheim Pharmaceuticals
- Invitae
- Dohmen Life Science Services
- Sobi
- AllianceRx Walgreens Prime
- Massachusetts Biotechnology Council (MassBio)
- HPS Group LLC.
- BioNJ, Inc.
- Biotechnology Industry Organization (BIO)
- Zafgen
- Vital Therapies
- Stealth Biotherapeutics
- Sarepta Therapeutics
- Insmed Incorporated
- GlycoMimetics, Inc.
- Cytokinetics, Inc.
- Strongbridge Biopharma
- Clementia Pharmaceuticals, Inc.
- Catalyst Pharmaceutical Partners
- Asklepion Pharmaceuticals, LLC
- Amicus Therapeutics, Inc.
- Vertex Pharmaceuticals Worldwide
- Soligenix, Inc.
- Shire Human Genetic Therapies, Inc.
- Retrophin, Inc.
- Recordati Rare Diseases
- Pfizer, Inc.
- Otsuka America Pharmaceutical, Inc.
- Novartis
- Takeda Oncology
- Lundbeck, Inc.
- Johnson & Johnson Pharmaceuicals
- Jazz Pharmaceuticals PLC
- Incyte Corporation
- Horizon Pharma
- GlaxoSmithKline PLC
- Sanofi Genzyme
- Genentech, Inc.
- CSL Behring
- Celgene Corporation
- Biogen
- BioMarin Pharmaceuticals, Inc.
- Alexion Pharmaceuticals, Inc.
- Aegerion Pharmaceuticals, Inc.
- Actelion Pharmaceuticals
- AbbVie Pharmaceuticals
- APCO WorldWide
- Agios
Physician Guides
- Pigmented Villonodular Synovitis
- Urea Cycle Disorders (UCD)
- Pompe Disease (Glycogen storage disease, type II; Acid maltase deficiency)
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Nontuberculous Mycobacterial Lung Disease (NTM)
- Myleofibrosis
- Mitochondrial Myopathy (MM)
- Lipoprotein Lipase Deficiency (LPLD)
- Lipodystrophy Disorders
- Infantile Spasms
- Hepatocellular Carcinoma (HCC)
- Acute Myeloid Leukemia (AML)
- Homozygous Familial Hypercholesterolemia (HoFH)
- Gaucher Disease
- Cutaneous T-Cell Lymphomas (CTCL)
- Atypical Hemolytic Uremic Syndrome (aHUS)