Patients & Caregivers
Patient Organizations
Clinicians & Researchers
NORD en Español
Contact NORD
We're celebrating
40 years!
Donate
Rare Disease News
Resource Library
About Us
Events
Understanding Rare Disease
Where to start
Rare Disease Facts and Statistics
NORD’s Rare Disease Database
Rare Disease Video Library
What It Means To Be Undiagnosed
Find A Rare Disease Organization
Stay informed
Stories That Inspire
RareEdu
®
– Online Learning Platform
Rare Disease Day
Resource Library
Publications On Rare Disease
Patient stories
Christina’s Lease on Life
Read More
Living with a Rare Disease
Manage your care
Getting Help & Support
Managing Your Disease
We can help
How NORD Can Help
Speak To Someone at NORD
Rare Disease Centers Of Excellence
Patient Assistance Programs
Explore Clinical Trials
Find A Patient Organization
Caregiver Resources
You Are Not Alone.
Resources & Services
Learn More
Community Support
Where to start
Rare Diseases Defined
Financial & Medical Assistance
Call Center & Information Services
Bringing Together Your Community
Mentoring organizations
NORD Member List
Start a Rare Disease Organization
Membership Program
Becoming Research Ready
Patient-Focused Drug Development
Improving clinical care
Rare Disease Centers of Excellence
Continuing Medical Education (CME)
Partnering with the community
Corporate Council
National Partnerships
Global Partnerships
Diversity, Equity & Inclusion
Community Support
Together We Are Strong.
Learn More
Advancing Research
For patients
List of Rare Diseases
Gene Therapy for Rare Disease
Find Clinical Trials & Research Studies
For researchers
Request for Proposals
Research Grant Programs
Data Standards for Rare Diseases
For clinicians
Resources for Patients
Find a Rare Disease Care Center
Continuing Medical Education (CME)
Connect
IAMRARE
®
Program Powered by NORD
Rare Disease Cures Accelerator (RDCA-DAP)
Add Your Expertise
Featured publication
Accelerator Pathway report
View More
Driving Policy
NORD policy and you
Public Policy Positions
Policy Statements & Letters to Policymakers
Rare Disease Advisory Councils
NORD’s State Report Card®
Taking action
Join the Rare Action Network®
Policy & Advocacy Taskforce
Take Action on Key Issues
Join A Current Campaign.
Learn about our current policy goals
Take Action
Get Involved
Raising Awareness & Funds with NORD
Do-It-Yourself NORD Fundraiser
Students for Rare
Sports & Fitness Fundraisers
Media Inquiries
In your community
Attend An Upcoming Event
Find a Rare Disease Patient Organization
Stay Informed With NORD’s Email Newsletter
Rare Disease Day®
Patient stories
Share Your Story
Careers At NORD
Intern At NORD
Jobs At Patient Disease Organizations
Corporate Council
About the Corporate Council
Corporate Council Members
Join Corporate Council
Code of Conduct
Show Your Support
Donate to NORD
Volunteer with NORD
Visit the NORD Store
Search for:
Donate
Understanding Rare Disease
Where to start
Rare Disease Facts and Statistics
NORD’s Rare Disease Database
Rare Disease Video Library
What It Means To Be Undiagnosed
Find A Rare Disease Organization
Stay informed
Stories That Inspire
RareEdu
®
– Online Learning Platform
Rare Disease Day
Resource Library
Publications On Rare Disease
Patient stories
Read More
Living with a Rare Disease
Manage your care
Getting Help & Support
Managing Your Disease
We can help
How NORD Can Help
Speak To Someone at NORD
Rare Disease Center Of Excellence
Patient Assistance Programs
Explore Clinical Trials
Find A Patient Organization
Caregiver Resources
You Are Not Alone.
Read More
Community Support
Where to start
Rare Diseases Defined
Financial & Medical Assistance
Call Center & Information Services
Bringing Together Your Community
Mentoring organizations
NORD Member List
Start a Rare Disease Organization
Membership Program
Becoming Research Ready
Launching Registries & Natural History Studies
Patient-Focused Drug Development
Improving clinical care
Rare Disease Centers of Excellence
Continuing Medical Education (CME)
Partnering with the community
Corporate Council
National Partnerships
Global Parnerships
Diversity, Equity & Inclusion
Community Support
Learn More
Advancing Research
For patients
List of Rare Diseases
Gene Therapy for Rare Disease
Find Clinical Trials & Research Studies
For researchers
Request for Proposals
Research Grant Programs
Data Standards for Rare Diseases
For clinicians
Resources for Patients
Find a Rare Disease Care Center
Continuing Medical Education (CME)
Connect
IAMRARE
®
Program Powered by NORD
Rare Disease Cures Accelerator (RDCA-DAP)
Add Your Expertise
Featured publication
View More
Driving Policy
NORD policy and you
Today’s Policy Issues
NORD’s Policy Statements
Rare Disease Advisory Councils
NORD State Report Card
Taking action
Join the Rare Action Network®
Policy & Advocacy Taskforce
Contact your Representative
Take Action on Key Issues
Join A Current Campaign.
Learn about our current policy goals
Get Involved
Raising awareness and funds with NORD
Do-It-Yourself NORD Fundraiser
Students for Rare
Sports & Fitness Fundraisers
Media Inquiries
In your community
Attend An Upcoming Event
Find a Rare Disease Patient Organization
Stay Informed With NORD’s Email Newsletter
Rare Disease Day®
Patient stories
Share Your Story
Careers At NORD
Intern At NORD
Jobs At Patient Disease Organizations
Show your support
Donate to NORD
Volunteer with NORD
Visit the NORD Store
Rare Disease News
Resource Library
About Us
For Clinicians & Researchers
For Patient Organizations
Home
/
Rare Disease News
/
Uncategorized
/
Living Rare, Living Stronger Forum
Set Valu
Keyword Search
Categories
Select an option
#NORDintheNews
Advocacy
Advocacy>Press Releases
COVID-19
Events
Featured News
Get Involved
Head of the Herd
Head of the Herd
Industry
Medicaid
Medical
Medical Affairs
Members
Newborn Screening
NORDPod
Partnerships
Partnerships>Rare Cancer Coalition
Partnerships>RDCA-DAP
Patient Stories
Patient Stories>Voices of Rare Cancer
Press Releases
Press Releases
Rare Cancer Coalition
Rare Disease Day
RareInsights
RareLaunch
RDACs
RDCA-DAP
RDCA-DAP
Registries
Research
Research>Registries
State Report Card
Sticky Posts for Homepage
Students for Rare
Uncategorized
Voices of Rare Cancer
Voices of Rare Cancer 2020
Tags
Select an option
{eter L. Saltonstall
#1in10
#1in10 Campaign
#BLM
#DoYourShare
#LightUpForRare
#LivingRareForum
#MissAmericaVote
#NORDGala15
#NORDinthenews
#NORDReport
#NORDSummit
#ProtectPatientsNow
#RareCancerDay
#RareDiseaseDay
#RareDiseases
#RareERChat
#RareImpact
#RDD18Chat
#SaveCHIP
#SaveOrphanDrugs
#ShowYourRare
#ShowYourStripes
#SSANDFORUM
#TCSNYCMarathon
000 Mile Rare Movement
1 in 10
100 Days Agenda
1983 Orphan Drug Act
2015
2016
2018 Rare Impact Award Honoree
2018 Rare Summit
2018 State Report Card
2019
2019 NORD Rare Summit
2019 Official Rare Disease Day Video
2019 Rare Impact Award Honoree
2020 Living Rare
2021
2022 NORD Rare Diseases and Orphan Products Breakthrough Summit
2022 Rare Disease and Orphan Products Breakthrough Summit
21st Centure Cures
21st Century Cures
21st Century Cures Act
22q11.2 deletion syndrome
2q23.1 Microdeletion/Duplication
2seventy bio
3-D printing
30 million
35 ways and growing
35 Years and Growing
35th Anniversary
35th Anniversary Celebration
35th Anniversary Celebration presenting the Rare Impact Awards
5 Myths
50 states
501(c)(3)
5K
7
7000 Mile Rare Movement
A Cure for Ellie
A Cure in Sight
A Short Walk Home
AA amyloidosis
AAC device
AAMDS
AAMDS International Foundation
AAMDSIF
Aaron Hamvas
aarskog syndrome
Abbey Meyers
Abbey S. Meyers
Abbey S. Meyers Leadership Award
Abby Norman
ABC News
ABCG5
abcg8
Abecma
Abeona Therapeutics
Abetalipoproteinemia
Abortion
ACA
ACA Individual Mandate
Academy of Country Music
Academy of Country Music Lifting Lives Music Camp for People with Williams Syndrome
accelerated approval
accelerated approval pathways
Accelerated Approval Program
Access
access to treatments
accessibility
ACD
ACD/MPV
ACDMPV
ACIP
ACIS
ACPMP
ACPMP Research Foundation
Acting Commissioner
Action Potential Grant
Acute lymphocytic leukemia
acute myeloid leukemia
acute myeloid lukemia
ADA
Adam Rose
Adaptive Marketing
adaptive yoga
adherence
adrenoleukodystrophy
Adriano Aguzzi
Adult Polyglucosan Body Disease (APBD) Research
Adult Rare Disease Patient
Advanced Premium Tax Credits
Advances in Pulmonary Hypertension
advancing drug development
Advancing Hope Act
Advancing Hope Act of 2016
Advancing Hope Act of 2016 (S.1878)
Advancing Innovation
Advancing Research for Neurological Diseases Act (S. 849)
Advancing Targeted Drugs for Rare Diseases Act
Advancing Targeted Therapies for Rare Disease Act
Advisory Committee on Immunization Practices
advocacy
advocacy alert
Advocacy Events
advocacy organizations
Advocate
affordability
Affordable Care Act
Affordable Care Act marketplace
afibrinogenemia
Agency for Healthcare Research and Quality
Agios
AGMD
AHCA
AHDS
aHUS
AI
AIDS Institute
Aileen Tobin
Aimee Guarnieri
AKU Society
ALD
Alex Azar
Alexa Moore
Alexander Pham
Alexandra Freitas
Alexion
Alexion Pharmaceuticals
ALIBER
Alison and Paul Frase
Alison Rocket Frase
Alison Rockett Frase
Aliza
Aliza Fink
ALL
All Things Kabuki
Allan-Herndon-Dudley syndrome
Alliance for a Stronger FDA
Alliance for Aging Research
Alliance for Connected Care
Allison Lint
alnylam pharmaceuticals
ALPS
ALS
ALS Association
Alston & Bird
alveolar capillary dysplasia
Alveolar Capillary Dysplasia Association
Alveolar Capillary Dysplasia with Misalignment of the Pulmonary Veins
Alynylam Pharmaceuticals
Ambassador
Ambry Genetics
AMENSupport
American Academy of Pediatrics
American Behcet's Disease Association
American Cancer Society
American Cancer Society Action Network
American Cancer Society Cancer Action Network
American College of Cardiology
American College of Medical Genetics and Genomics
American Health Care Act
American Healthcare Act
American Heart Association
American Lung Association
American Medical Student Association
American Medical Students Association
American Multiple Endocrine Neoplasia
American Multiple Endocrine Neoplasia (AMEN) Support group
American Patients First
American Plasma Users Coalition (A-PLUS)
American Porphyria Foundation
American Public Health Association
American Society of Hematology Annual Meeting
American University of Integrative Sciences School of Medicine
Americans with Disabilities Act
Amgen
amicus brief
Amicus Therapeutics
amniotic band syndrome
AMSA
Amyloidosis
Amyloidosis Support Groups Inc.
amyotrophic lateral sclerosis
Anagralide
and The Physician’s Guide series of booklets
and Treatment in the US: A 30-year Comparative Analysis
Andrew W. Mellon Auditorium
Angelia Benson
Angelman Syndrome
Ann & Robert H. Lurie Children’s Hospital of Chicago
annual report
Annual Report 2021
Anthem
Anthem Foundation
anthrax
anti-discrimination
anxiety
Apellis
Apellis Pharmaceuticals Inc
APF
Aplastic Anemia and MDS International Foundation
appendix
Appendix Cancer
Appendix Cancer and Pseudomyxoma Peritonei
Appendix Cancer/Pseudomyxoma Peritonei Research Foundation
approval standards
APS Type 1
APS Type 1 Foundation
APS-1
arachnoiditis
arginase deficiency
Arlington
Armed Services Committee
Art Alexakis
art exhibit
Arthritis Foundation
arthrogryposis
Arthur Caplan
ASGCT
ASH
Asklepion Pharmaceuticals
Assembly Education Committee
Associate Director for Strategic Initiatives
Association for Accessible Medicines
Association of Gastrointestinal Motility Disorders Inc.
Astellas Pharma
asthma and allergy foundation of america
AstraZeneca
Atara
ataxia
Athlete
atrial septal defect
Atypical HUS
Audrey Hepburn
August
autoimmune disease
Autoimmune Lymphoprolifertive Syndrome
autoimmune neutropenia
Autoimmune Polyglandular Syndrome Type 1
autonomic dysfunction
autosomal genetic disorder
Avalere
Avalere Health
AveXis
award
award nominations
Awards Ceremony
Aware of Angels
awareness
awareness day
Ayvakit
Azar
Baby Formula
Baby's First Test
Bailey Gribben
balance bill
BAM Syndrome
Banner on the Moon
Barb Calhoun
Barbara Hollister
barriers
Barriers to Rare Disease Diagnosis
Batten Disease
BBB
BBBA
BCRA
BDSRA
Be a Hero
Beau Biden
Beckwith-Weidemann Syndrome
Behcet's disease
Behind the Mystery
Believe Limited
bequest
Bespoke Gene Therapy Consortium
Besremi
Best Pharmaceuticals for Children program
beta-thalassemia
Beth Nguyen
Better Care Reconciliation Act
Better Care Reconciliation Act of 2017
Beverley Francis-Gibson
BGTC
bicycle
Bilary Atresia
bile duct cancer
biliary atresia
Bill
Bill Erickson
Billy Dunn
BIO
biobank
bioengineering
Biogen
biological pathways
biologics
BioMarin
BioMarin Pharmaceutical
BioMarin Pharmaceutical Inc
BioNews Service
BioNJ
BioNTech
bioprinting
biosimilars
Biotechnology Industry Organization
bioterrorism
birth defect
Black Bone Disease
black lives matter
Black Women’s Health Imperative
Block Grants
Blood
blood cancer
blood cancer awareness month
blood counts
Blood Disorders
bluebird bio
Blueprint
Blueprint Medicines
BoardSource
Bob Saget
Boehringer Ingelheim
Boler-Parseghian Center for Rare and Neglected Diseases
bone cancer
bone marrow
bone marrow transplant
Bonnell Foundation
Bonnie Royster
book
Book of Hope
Bosma arhinia microphthalmia (BAM) syndrome
Boston Children's Hospital
Boston Marathon
Boston Marathon 2015
Boston Medical Center
brain
Brain Cancer
Brain Tumor
Brandon Hudgins
Brandstar
Breakthrough Summit
Breakthrough Therapy Designation
breast cancer
Brent Clough
Brian Kemp
Bridge the Gap-SYNGAP Education and Research Foundation
BridgeBio Pharma
Bristol Myers Squibb
Brugge Breast Cancer Lab
Budapest
Build Back Better
Build Back Better Act
bullying
Bustle
C-Path
CA
Cabergoline
CACNA1A
California
California Attorney General Xavier Becerra
Call Center
Call for Posters
Calliope Joy Foundation
Cancer
Cancer in our Genes Patient Databank
Cancer Moonshot
Cancer Support Community
cannabinoids
capacity-building
Capitol Hill
CARD9 Deficiency
cardiac
cardiology
cardiomyopathy
Care
caregiver
Caregiver Respite
caregivers
Caring for Carcinoid Foundation
Carmina Taylor
Carolyn Sheridan
CAT
Cat Eye Syndrome
CAT Scan
Catalyst Pharms.
catecholamines
Catherine Blansfield
Cathleen Lutz
Cavernous Angioma
CBER
CBO
CBS News
CCF
CCHS
CCHS Family Network
CCHS Foundation
CCPA
CCS
CDC
CDER
CDER’s Associate Director for Strategic Initiatives
CdLS
CdLS Foundation
CDRH
celebration
Celera Genomics
Center
Center for Biologics Evaluation and Research
Center for Drug Evaluation and Research
Center for Drug Evaluation and Research (CDER)
Center for Innovation in Global Health
Center for Responsible Science
Center of Excellence
Centers for Disease Control
Centers for Disease Control and Prevention
Centers for Medicare and Medicaid Services
centers of excellence
central congenital hypoventilation syndrome
centronuclear
cerebral palsy
Certified Nursing Assistant
Chagas
Chalk the Walk
CHAMP1
Chan Zuckerberg Initiative
Chandra Clark
Change Together
Charity Navigator
Charity of the Week
Charity Partner
charity running team
Charlene Son Rigby
Charlene York
Chasten Buttigieg
chemo
chemo brain
chemotherapy
CHI
Chiari Malformation
Chicago
Chicago Illinois
chief medical and scientific officer
childhood cancer
Children's Cardiomyopathy Awareness Month
Children's Cardiomyopathy Foundation
Children's Health Insurance Program
Children's Hospital of Los Angeles
Children's Hospital of Philadelophia
Children's Hospital of Philadelphia
Children's Inn
Children's National
Children's National Director
Children's National Health System
Children's National Hospital
Children's National Medical Center
Children’s Cardioymyopathy Foundation
Children’s Connecticut Medical Center
Children’s Hospital and Hackensack Meridian School of Medicine
Children’s Miracle Network
CHIP
CHOC Children’s
CHOC Children’s Hospital
cholangiocarcinoma
Cholangiocarcinoma Foundation
Chondrosarcoma
CHOP
CHOP Leukodystrophy Center
chris anselmo
Chris Brannigan
Chris Ulmer
Christine Grube
Christopher Anselmo
chromosome 16
chronic disease
Chronic Disease Coalition
chronic Epstein-Barr virus (EBV)
Chronic Granulomatous Disease
Chronic Illness
chronic inflammatory demyelinating polyneuropathy
chronic migraine
chronic pain
Chung Lab
Cicatricial Alopecia Research Foundation (CARF)
CIDP
CIDP Foundation
CIDP Foundation International
Cincinnati Children's Hospital Medical Center
Cindy Abbott
Citizen Petition
Citizen's Petition
CL Foundation
Claude Steriade
clear cell sarcoma
Cleveland
Cleveland Clinic
Clinic for Special Children
clinical trials
CLOVES Syndrome
CLOVES Syndrome Community
CME
CMS
CMTC-OVM
CNA
CNM
CNTNAP1 mutation
CO
co-pays
Coalition
Coalition Statement
Coats Plus Syndrome
Cockayne Syndrome
CoE
Coffin-Siris Syndrome
collaboration
Colleen King
Colonel Ken Cameron
colorado
Columbia Presbyterian
Columbia University Irving Medical Center
Columbia University Medical Center
Columbia University’s Fu Foundation School of Engineering
COMBINEDBrain
Commissioner
Commissioner of the FDA
community
community survey
Community Survey Report
compassionate allowances
Compassionate Use
compliance
computer
computer-assisted diagnosis
Conduct of Clinical Trials of Medical Products during COVID-19 Public Health Emergency
conference committee
congenital central hypoventilation syndrome
congenital heart defects
congenital hyperinsulinism
Congenital Hyperinsulinism International
congenital muscular disorder
congenital muscular dystrophy
Congress
Congressional Budget Office
Congressional Childhod Cancer Congress
Congressional Leadership
Congresswoman Doris Matsui
Congresswoman McMorris Rodgers
Congresswoman Robin Kelly
Connecticut
Connecticut magazine
Connecticut-based nonprofits
Connecting ALS
Constitution
Consumer Assistance Program
continuing medical education
Continuing Resolution
contracture
COPA Syndrome Foundation
copay accumulator programs
copay accumulators
COPPA
CORD
Cornelia de Lange Syndrome
Cornelia de Lange Syndrome Foundation
coronavirus
cost of orphan drugs
cost-sharing
Council of National Alliances
Court
Court Case
coverage
covid
covid-19
COVID-19 Premium & Medical Relief Program
COVID-19 Rapid Response Leadership Series
covid19
CR
Craine’s Cholangiocarcinoma Crew
Creating Hope Act
Creutzfeldt-Jakob Disease
crisis communications
crisis management
CRISPR
Cristina Y. Go MD
Critical Path
Critical Path Institute
CRMCC
CRN Family Conference
Crohn’s disease
Crowdfunding
CSS
CTCL/MF
CTF
Cure CMD
Cure MLD
Cure SMA
CureDuchenne
CureDuchenne Cares
CurePSP
CuresNow
Curing Retinal Blindness Foundation
Cushing's Disease
cutaneous lymphoma
Cutaneous Lymphoma Foundation
cutaneous T-cell
Cutis Laxa
Cutis Laxa Internationale
cutis marmorata telangiectatica congenita
cyclodextrin
Cystic Fibrosis
Cystic Fibrosis Foundation
Cystic Fibrosis Research Inc.
Cystinosis
Cystinosis Research Network
Cytalux
CZI
Dad
Daiichi-Sankyo
Dana-Farber Cancer Institute
Daniel Stanley
Daniela Delgado
Daniele Batlle
Danny's Dose
Danyelle Sun
Data
Data-Sharing
DataRevive LLC
Davelyn Hood
David Arons
David Cry
David Geffen School of Medicine at UCLA
David Lebwohl MD
David Meeker
Dawn Laney
day of action
Days of Action to Protect Your Heatlhcare Coverage
DC
deafness
Deanna Portero
Debbie Drell
Debbie Skolaski
Deborah Skolaski
DEBRA International
Debra Miller
Debra of America
Debra Regier MD PhD
DEI
Delaware
Dennis Jackman
Department of Health & Human Services
Department of Health and Human Services
Department of Insurance
dermatology
Desiree Lyon
Desmoid Tumor Research Foundation
development
developmental delay
Devic syndrome
Devin Alvarez
DHHS
DHPS
DHPS Foundation
DHPS Patient Registry
diabetes insipidus
diabetes mellitus
Diagnosis
diagnostic odyssey
Diagnostic Testing
diagnostician
diagnostics
Diane Dorman
diaphoresis
Diazoxide
DiGeorge syndrome
Digestive Motility Disorders
digital health
DIPG
direct-to-consumer genetic testing
Director of Marketing and Communications
Director of Membership
Director of Patient Services
Director of Research
Director of the Center for Drug Evaluation and Research at the Food and Drug Administration
Director of the Center for Thoracic Insufficiency Syndrome
Director Walensky
Disabilities
disaster relief for rare disease patients
disease
disease-specific Emergency Relief Programs
Disney
distal 18q deletion
Diversity
Division Chief of Genetics and Metabolism
Division of Endocrinology at Northwestern
DMD
DNA
DNMT1 gene
DNMT3A
Dobbs v Jackson
Doctorate of Nursing Practice
doctors
documentary
Dolly the sheep
Dompe
Donate
Donate to NORD
donation
Donna Appell
Donna Appell RN
Download
Dr. Amanda Cohn
Dr. Anita Gupta
Dr. Christoper Austin
Dr. Christopher Austin
Dr. Christopher Klein
Dr. Cynthia Tifft
Dr. Diego Fornasari
Dr. Donald C. Vinh
Dr. Eli Diamond
Dr. Francis S. Collins
Dr. Frederick Kaplan
Dr. Janet Woodcock
Dr. John Graham
Dr. Juvianee Estrada-Veras
Dr. Katherine Leaming
Dr. Levy
Dr. M Kathryn Liszewski
Dr. Margaret Hamburg
Dr. Marshall Summar
Dr. Marshall Summars
Dr. Marty Makary
Dr. Melinda Burnworth
Dr. Michael F. Goldberg
Dr. Michelangelo Mancuso
Dr. Michio Hirano
Dr. Natalie Shaw
Dr. Noonan
Dr. Pantea Sharifi Hannauer
Dr. Peter Marks
Dr. Rachel Harper
Dr. Richard Besser
Dr. Richard Pazdur
Dr. Robert Califf
Dr. Robert Campbell
Dr. Robert Coleman
Dr. Robert M. Campbell
Dr. Scott Gottlieb
Dr. Stephen Hahn
Dr. Stephen Ostroff
Dr. W. French Anderson
Dr. Wendy Chung
Dr. Yeboa
draft guidance
Dravet and Hurler syndromes
Dravet Syndrome
Dravet Syndrome Foundation
Drexel University
drug costs
drug development
drug development process
drug discovery
Drug Manufacturers
Drug Prices
drug pricing
drug research
drug sales
dTRA
duchenne muscular dystrophy
Duct Tape Won't Fix This
Duke University
Dynata
dysarthria
dyslexia
Eallonardo Family
early diagnosis
Ebanga
EBRP
EBV
ECD Global Alliance
ECD Patient and Family Gathering
ECD Referral Care Centers
ECMO
Ectodermal dysplasia
Ed Silverman
Editas Medicine
Editorial Collaboration
EDS
Eduational Initiatives
Education
Educational Event
Educational Initiatives
educational video
Educational Webinar
edward neilan
Ehlers Danlos Syndrome
Ehlers-Danios Syndrome
Ehlers-Danlos
Eiger Biopharmaceuticals
EL-PFDD
Eleanor Perfetto
Eli Lilly
Elisabeth Dykens
Eliza Rista
Elizabeth Katz
Elizabeth Silva Anderson
Ellen V. Sigal
Ellie McGinn
Emergency Relief Resources Packet
Emergency Rom Visits
Emergency Room Protocol
emergency use authorization
Emily Argersinger
Emory University
Emory University Department of Human Genetics
Empaveli
End NF
endocrinology
Energy and Commerce Subcommittee
Energy for Life Walkathon
Ensuring Access to Clinical Trials Act
Entering a New Era
Enzyvant Therapeutics Inc
epidermolysis bullosa
Epilepsy Foundation
epileptic encephalopathies
Epstein-Barr virus
equality
equity
ER with a Rare Disease
Erdheim Chester Disease Awareness Week
Erdheim-Chester Disease
Erica Barnes
Ernst & Young
essential health benefits
Essential Thrombocythemia
estate
estate plan
Estelle Benson
ET
ethics
EUA
Eugene Washington Engagement Award
EURODIS
European Commission
European Union
eurordis
EURORDIS-Rare Diseases Europe
Eva Juneja Foundation
Eva's Butterfly Wishes for Walker-Warburg Families
Event
Events
Eversana
Eversource Hartford Marathon
EveryLife Foundation for Rare Diseases
Expanded Access
externally led Patient-Focused Drug Development
externally-led Patient-Focused Drug Development Meeting
extracorporeal membrane oxygenation
EY
Eye Believe Patient Survivorship Meeting
Fabry Disease
Facebook
Facebook Live
factor-vii deficiency
Familial Hypercholesterolemia
Families
FamilieSCN2A Foundation
family
Fanconi Anemia
Fanconi Anemia Research Fund
FAQ
FARF Translational Science Director
Father
Father's Day
FD/MAS Alliance
FDA
FDA Acting Director
FDA CDER
FDA Commissioner
FDA Commissioner Stephen Hahn
FDA Matters
FDA OCE
FDA Oncology Center of Excellence
FDA Patient Affairs
FDA Public Meeting
FDA Rare Diseases Program
FDA Reauthorization Act
FDA Reauthorization Act of 2017
FDA Safety & Innovation Act
FDA Safety and Innovation Act
FDA user fees
FDARA
FDASIA
Febrile Infection-Related Epilepsy Syndrome
February 28
February 28th
February 29
federal government
federal legislation
federally regulated insurance
fibrolamellar
Fibrolamellar Cancer
Fibrolamellar Cancer Foundation
fibrolamellar carcinoma
fibromyalgia
financial assistance
financial support
Financial Times
Finding a Zebra Among Many Horses
FIRES
FIRST
Florida
Food & Drug Administration
Food and Drug Administration
Food and Drug Administration Reauthorization Act of 2017
Food and Drug Amendments of 2022
FOP
Forming a Foundation
Formula
Formula Shortage
formulary
Foundation for Ichthyosis and Related Skin Types
Foundation for Prader-Wili Research
Foundation for Prader-Willi Research
Foundation for Rare Blood Diseases
Foundation to Fight H-abc
FOXF1
FOXF1 gene
FPIES Foundation
Frances Flanagan
Francis Collins
Frank Sasinowski
Fred Upton
Free Will
FreeWill
Friedreich's ataxia
Friedreich’s Ataxia Research Alliance
Friends of Cancer Research
From Emotions to Advocacy by Pam Wright and Pete Wright
Frontline Medical Communications
Full House
Fundraiser
fundraising
Gabriel Low
Gain Therapeutics
galactocerebrosidase
galactosemia
Galactosemia Foundation
GALC
GAN
GARD
Garrett Mann
gastroenterology
gastrointestinal stromal tumors
Gastroschisis
Gaucher Disease
GBS
GBS CIDP Foundation
GBT
GDPR
Gene
gene editing
gene mutation
gene replacement therapy
Gene therapy
Genentech
generics
genes
Genet Testing
Genetech
genetest101
genetic
Genetic and Rare Diseases Information Center
genetic biobanks
Genetic Counselor
genetic disease
genetic disorder
genetic testing
Genetics
genome editing
genomic testing
Genzyme
George Soros
Georgetown School of Medicine
Georgia
Get Involved
Get Invovled
gift
GIST
Give Kids the World
Giving Spotlight
Giving Spotlight. Giving to NORD
Giving to NORD
Givlaari
GlaxoSmithKline
Glenn S. Williams
Global
Global Blood Therapeutics
Global Chain of Lights
Global Foundation for Peroxisomal Disorders
Global PNH Patient Registry
Global Video
globoid cell leukodystrophy
Glybera
Good Governance
Good Morning Peyton
Gorlin Syndrome
Gorlin Syndrome Alliance
government
governor
Governor Jared Polis
Governor Kemp
Governor Matt Bevin
Governor of Kansas
governors
Grace Rista
Graham-Cassidy
Graham-Cassidy Bill
grant
grants
Guadalajara Mexico
Guest Blog
Guest Blog Post
guest blogger
Guillain-Barre syndrome
Guillan-Barré syndrome
Guthy-Jackson Foundation
GW Pharmaceutics
gymnastics
H-abc
H.R. 3783
H.R. 669
HAE
Hal Dietz M.D.
Hand Prints Across America
Handprints Across America
Hannah Kane
Hannah's Hope
Hao Cheng
Harjot Randhawa
Harvard Medical School
Harvard T.H. Chan School of Public Health
Hashimoto syndrome
HB239
Head of the Herd
Health
Health & Human Services
Health and Human Services
Health and Human Services Secretary
Health Coverage Reform
health crisis
health disparities
health disparity
health equity
health insurance
health insurance marketplaces
health programs
healthcare
Healthcare Bill
healthcare costs
Healthcare Coverage
healthcare reform
healthcare.gov
HealthDay
Healthline
Healthwell Foundation
Heart Disease
hEDS
Heidi Ross
HELP Committee
Helping Hands for GAND
hematology
hemophilia A
Hemophilia Federation of America
Henry Waxman
Hepatocellular carcinoma
hepatology
Hereditary Leiomyomatosism
Hereditary Neuropathy Foundation
Hereditary Sensory Autonomic Neuropathy Type 1E
Heriditary Angiodema
Hermansky Pudlak Syndrome
HFA
HHS
HHS Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs
HI
HI Global Registry
hidradenitis suppurativa
high-risk pool
Highlights Video
HIPAA
histiocytes
histiocytosis X
HLA B27
HLA B27 autoimmune disease
HLRCC Family Alliance
Home Health Aide
home infusions
HOME Study
HomeServe USA
Homocystinuria
Homocystinuria due to Cysathionine Beta-Synthase Deficiency
Hope for Hasti
Horizon
Horizon Therapeutics
House
House Energy & Commerce
House of Representatives
House Ways and Means Committee
Houston
Houston TX
How Full Is Your Bucket?
HPP
HPS
HPS Group
HPS Network
HR 7667
HSAN1E
HSAN1E Society
Human Genome Project
Humanitarian Device Exemption
hunger
Hunter's Hope
Huntersville North Carolina
Huntington's disease
Huntington’s Disease Youth Organization
hurricane dorian
Hurricane Harvey
hurricane ian
hurricane relief
Hyper IgM Foundation
hyper IgM syndrome
hyperphagia
hypoglycemia
hypokalemic periodic paralysis
hypophosphatasia
Hypoplasia
hypotonia
hypoxemia
Hyptonia
I-ORD
IACT
IAMRARE
IAMRARE Patient Registry
IAMRARE Registry
IAMRARE Registry Program
IBM
IBM Watson
ICD Codes
ICER
Ichthyosis
Ilene Sussman
Illinois
Illinois Rare Disease Commission
Immune Deficiency Foundation
immune system
immunoglobulin
immunosuppressed
Impact
Improving Access to Clinical Trials Act
inborn errors of metabolism
Inc.
Inc. v. Becerra
inclusion
Inclusivity
inclusivity policy
Incyte
Incyte Pharmaceuticals
industry
Infantile Myofibromatosis.
Infantile Spasms
infection
infectious disease
Inflation Reduction Act
influencer
infographic
informed conset
infusion coverage
Ingo Helbig
inherited metabolic disorders
Inmazeb
Innovative Research
INSIGHT
INSIGHT: A Global Ocular Melanoma Patient Registry
Insmed
Insmed Inc.
Inspiration
insurance
insurance caps
insurance market protections
insurance-like products
Interferon
internal medicine
International Family Registry for Centronuclear and Myotubular Myopathies
international rare disease day
International Spy Museum
intracranial hypertension
intrahepatic cholangiocarcinoma
Iowa State University
IQVIA
IQVIA Institute
IV infusion therapy
IVQIA
Jack Klugman
Jack Reed
Jacob Haendel’s Recovery Channel
Jacqueline Kraska
Jade Day
Jakafi
Jane Larkindale
Janet Bunge
Janet Woodcock
Janet Woodcock MD
Jann Le Cam
Jansen's
Jansen's Foundation
Jared Polis
Jason Sicklick
Jayne Holtzer Rare Disease Research Grants Program
JDRF
Jefferson University Medical College
Jeffersonian dinner
Jennifer Canvasser
Jennifer Orange
Jennifer's GP House
Jeopardy
Jess Thoene
Jessica Bohonowych
Jill Pollander
Jill Zeigler
Jim Greenwood
Jim Palma
Jimmo v. Sebelius
Jiping Yue
Joe Biden
Joe DiMaggio Children’s Hospital
John Farley
John Hopper
John Jenkins
John K. Jenkins
John Kline
John McCain
John Oliver
John Rista
Johnny Rista
Johns Hopkins Medicine
Johns Hopkins University School of Medicine
Jonathan C. Goldsmith
Jordans Syndrome
José Abdenur
Joseph Farris
Joseph Scheeren
Joseph Shieh
Joshua Frase
Joshua Frase Foundation
joshua mann
journal
JPA
Jr. & Georgia O. Akers Private Foundation
Julia Su
Julia Vitarello
Julie Raskin
Juvenile Rheumatoid Arthritis
Kabuki syndrome
Kam Redlawsk
Kansas Department of Health and Environment
Karen Pignet-Aiach
Kari Luther Rosbeck
karina sturm
KAT6A Foundation
KAT6A Patient Registry
KAT6A syndrome
Kate Bartlett
Kate Obstgarten Family Foundation
Katie Larimore
Katie Thortenson
Kawasaki disease
Kay Holcombe
Keck Graduate Institute
Kedrion P BioPharma Inc.
Kelly Barendt
Kentucky
Kerri Nelson
keynote
Keynote Address
Keynote Speaker
Kira Mann
Kjersti Flatmark
Klaus Romero
Koselugo
krabbe
Krabbe Community United Research and Engagement Study
Krabbe disease
KrabbeConnect
KrabbeCURES
Kristen Angell
Kristin Anthony
Kristin Smedley
KY3
LAM
Langerhans Cell Histiocitosis
Langerhans cell histiocytosis
Lankenau Medical Center
Laura Crandall
Lauren Hill
Lauren Ruotolo
Lauren Wasser
LBSL
LD 972
Leah Lande
leap day
Learning
leber’s hereditary optic neuropathy
Legg Calves Perthes Foundation
legislation
Legislative Document 972
Leigh Syndrome
leiomyosarcoma
Lennox-Gastaut
Lesli Nordstrom
leukemia
Leukemia & Lymphoma Society
Leukemia and Lymphoma Society
leukodystrophy
Levy-Yeboa Syndrome
Levy-Yeboa Syndrome Research
Lewis & Clark College
Lexington Clinic
lgbtq+
LGDA
Li Fraumeni Syndrome
Li Fraumeni Syndrome Association
Liam Cruz
Licensed Practical Nurse
life sciences
Life-Transforming Treatments
Lifetime Achievement Award
Lifetime Television
Light Up for Rare
limited medical assistance
Lindsey Graham
lipidosis
Lipoprotein Lipase Deficiency
Lipoprotein(a) Foundation
Lisa Butler
Lisa Phelps
Lisa Sarfaty
Lisa Terrizzi
Lisa Yue
Listening Sessions
Live Webcast
livestream
Living Rare
Living Rare Forum
living rare living strong
Living Rare Living Stronger
Living Rare Living stronger Forum
Living Rare Living Stronger NORD Patient & Family Forum
Living Rare Living Stronger NORD Patient and Family Forum
living rare living stronger patient & family forum
Living Rare Living Stronger Patient and Family Form
Living Rare Living Stronger Patient and Family Forum
living strong
Living Stronger
Living Stronger NORD Patient and Family Forum
Living Stronger™ NORD® Patient a
Lizzie Valasquez
LMS
Logo
longitudinal
Los Angeles
Los Angeles Times
Lou Gehrig's disease
Louisiana
Loxo Oncology
LPLD
LRLS
LRLSR
Lundbeck
Lundbeck US
LungMAP
Lurie Children's Hospital
Lutheran Services in America
Lymphangioleiomyomatosis
Lymphangiomatosis & Gorham's Disease Alliance
Lymphedema
lymphoma
LYS
Lysogene
lysosomal disease
Lysosomal Storage Diseases
lysosomal storage disorder
lysosomal storage disorders
M.D.
M.S.c.
MA
MacArthur Amendment
Mackenzie's Mission
Maddie's Herd
Madison Shaw
Magenta Therapeutics
Maine
Make a Will
Make a Will Month
Make-A-Wish Foundation
Mallinckrodt Pharmaceuticals
Malonia Aciduria
Malonic Aciduria
MAPkinase pathway
Marathon
Marc Scheineson
March of Dimes
Marcy Toepel
Marfan syndrome
Margaret Hamburg
marginalized
marginalized communities
Maria Kefalas
Marion Finkel
mark anastasio
market reforms
Marketing Approval
Marketplace
Marsha Lanes
Marshall L. Summar
Marshall Summar
Martha Rinker
Mary Dunkle
Mary Lou Retton
Mary Wooten
Mary Wootten
Massachusetts
Massachusetts College of Pharmacy and Health Sciences
Massachusetts General Hospital
Mast Cell Disease
Matthew MIght
Matthew Pearl
matthew zachary
Mauli Ola Foundation
Maurice Klugman
Mayer-Rokitansky-Kuster-Hauser syndrome
mayo-clinic
MCADD
McGill School of Medicine
McGill University Health Centre Research Institute
MD
MD Anderson Cancer Center
MdDS Foundation
MDS
MDS Foundation
ME
Medal
Media
MediaPlanet
Medicaid
Medicaid eligibility
Medicaid enrollment
Medicaid expansion
Medicaid section 1115 waivers
Medicaid/CHIP eligibility
medical
Medical Advisory Committee
Medical Alert Bracelet
medical device
medical devices
Medical Education Advancement
Medical Ethics
Medical Foods
medical foods coverage
medical geneticist
Medical Nutrition
Medical Nutrition Equity
medical nutrition equity act
Medical Product Innovation
medical professionals
Medical research
medical resources
medical reviewers
Medical Students
medically diverse
Medicare
medication refills
medium-chain acyl-CoA dehydrogenase deficiency
Medscape
Megacystis Microcolon Intestinal Hypoperistalsis Syndrome
Melanie Langelier RN
Melvin Van Woert
Member Organizations
Member Spotlight
Members
Members-Only Webinar
Membership Manager
Membership Organizations
Memorial Sloan-Kettering Cancer Center
MEN
MEN-1
mended hearts
Menin
Mental Health
Merced
Mered Parnes
mesothelioma
metachromatic leukodystrophy
Michael A. Pack
Michael Rodgers
Michael Scott
Michael Smedley
Michael's Cause
Michelle Campbell
Mike Porath
Mike Scott
Mila’s Miracle Foundation
military
Milken Institute
Miss America
Miss Connecticut
Miss Iron County
Miss Pre-Teen Connecticut
Miss Utah
MitoAction
MitoAction Mobile
Mitochondrial Disease
Mitochondrial Disorders
Mitochondrial Myopathies
Miyoshi myopathy/limb-girdle muscular dystrophy type 2
MLD
MMA
MMIHS
MMN
Moderna
Mother's Day
Mount Everest
Mount Sinai Hospital
movement disorders
movies
moyamoya
MPN Research Foundation
MPN specialists
MPN-NET/MPN Education Foundation
MPNs
MPS I
MPS I Hurler syndrome
MPSI
MRI
MS
MSKCC
Mt. Washington
MTM
MTM1
Mucopolysaccharidosis IIIA
Mucopolysaccharidosis type 1
multicentric reticulohistiocytosis
multifocal motor neuropathy
Multiple Sclerosis Foundation
Muscular Dystrophy
Muscular Dystrophy Association
myasthenia gravis
mycosis fungoides
myelodysplastic syndromes
Myelofibrosis
myeloproliferative neoplasms
Myhre Syndrome
myositis
myotubular myopathy
Narcolepsy
Narcolepsy Network
narcolepsy type 2
NASA
NASA Astronaut
Nasogastric Tube
Natalia Gomez-Ospina
Natasha Shur
National Brain Tumor Society
National Cancer Institute
National Center for Advancing Translational Sciences
National Center for Advancing Translational Sciences (NCATS)
National Commission on Orphan Diseases
National Defense Authorization Act
National Down Syndrome Congress
National Family Caregivers Month
National Foundation for Ectodermal Dysplasias
National Fragile X Foundation
National Health Council
National Hemophilia Foundation
National Institues of Health
National Institute of Environmental Health Sciences
National Institutes of Health
National Institutes of Health (NIH)
National Institutes of Health (NIH) Undiagnosed Diseases Network (UDN)
National Institutes of Health Undiagnosed Diseases Program
national kidney foundation
National Leiomyosarcoma Foundation
National Marrow Donor Foundation
National MPS Society
National Multiple Sclerosis Society
National Organization for Rare Disorders
national patient advocate foundation
National Pediatric Research Network Act
National Psoriasis Foundation
National Rare Disease or Condition Surveillance System
National Urea Cycle Disorders Foundation
National Volunteer Week
Nationwide Children’s Hospital
Natpara
natural disaster
Natural Histories
natural history
natural history studies
natural history study
Natural History Study Data
Natural History Study Project
Navigating Pediatric ER
Navigating the Pediatric ER
nbs
NBTS
NCATS
NCC Collaborator
Nebraska
NEC
NEC Society
necrotizing enterocolitis
Ned Sharpless
Neena Nizar
NEHI
NEHI Research Foundation
Neil Horikoshi
neonatal intensive care unit
NET cancer
Network
Networking
neurodevelopmental disorders
neuroendocrine cancer
Neuroendocrine Cancers
Neuroendocrine Cell Hyperplasia of Infancy
Neuroendocrine Tumor Research Foundation
Neurofibromatosis Network
Neurofibromatosis Type 1
Neurogene
Neurogenic Orthostatic Hypotension
neurologic
neurology
Neurology Reviews
neuromuscular
Neuromyelitis Optica
Neuromyelitis Optica Spectrum Disorder
New England Journal of Medicine
New Jersey
New York City
New York City Police Department
New York Road Runners
New York University
New York University School of Medicine
New-Onset Refractory Status Epilepticus
newborn
newborn screening
Newborn screening awareness month
Newborn Screening Saves Lives Act
newly diagnosed
Next-Generation Treatments & Advancing Clinical Trials
nf1
NF2
NFL
NGLY1
Nibs
NICU
Niemann-Pick disease type C1
NIH
NIH Clinical Educator of the Year
NIH funding
NIH National Center for Advancing Translational Sciences
NIH research
NIH Resources
NIH Undiagnosed Diseases Program
NKH
NLMSF
NMO
NMO disease
NMOSD
Noah Victoria
nOH
Nominate
nominees
non-medical needs
non-orphan
non-orphan specialty drugs
non-profit leaders
Nonketotic Hyperglycinemia
Nonprofit
nonprofit organizations
nonprofits
Nontuberculous Mycobacterial Lung Disease
Noonan Syndrome
Noonan Syndrome Foundation
NORD
NORD 2022 Rare Disease and Orphan Products Breakthrough Summit
NORD 35th Anniversary
NORD 35th Anniversary Celebration
NORD Board
NORD Board of Directors
NORD COVID-19 Community Survey Report
NORD COVID-19 resource center
NORD Education Team
NORD Event
NORD Founder
NORD Hurricane and Natural Disaster Emergency Relief Fund
NORD IAMRARE Patient Registries
NORD in the News
NORD Medical Advisory Committee
NORD Member Organization
NORD Member Organizations
NORD Members
NORD Online
NORD Patient and Family Forum
NORD Patient Assistance Program
NORD Physician Guides
NORD Policy Team
NORD Position Paper
NORD RAN
NORD Rare Caregiver Respite Program
NORD Rare Disease Video Library
NORD Rare Diseases & Orphan Products Breakthrough Summit
NORD Rare Diseases and Orphan Products Breakthrough Summit
NORD Rare Summit
NORD registry community
NORD Research Grant
NORD Research Grant Program
NORD Resource Guide
NORD Resources
NORD Student Chapter
NORD Student Programs
NORD Students for Rare Club
NORD Summit
NORD survey
NORD Website
NORD's 35th Anniversary
NORD's 35th Anniversary Celebration
NORD's 35th Anniversary Celebration presenting the Rare Impact Awards
NORD's Medical Advisory Committee
NORD's Mission
NORD's Patient Assistance Program
NORD’s Jayne Holtzer Rare Disease Research Grants Program
NORDGivesBack
NORDpod
Norman E "Ned" Sharpless
Norman Winnerman
NORSE
Northeastern University
Northwestern Memorial Hospital
Northwestern University
Northwestern University Feinberg School of Medicine
Northwestern University School of Medicine
Notre Dame
Novartis
Novartis Oncology
Novimmune and Sobi
NTM
NTM Info & Research Inc.
NTM Research & Info
Nulibry
NYC
NYC Marathon
NYS Bill A1470B-2015
NYU
NYU Hospital for Cancer and Blood Disorders
NYU Langone Medical Center
NYU School of Medicine
O. Carter Snead III MD FAAN
Obamacare
obstetrics
Octavio Armenta
ocular melanoma
ODA
ODTC
odyssey
Office of Civil Rights
Office of Hematology and Oncology Products
Office of New Drugs
Office of Orphan Products Development
Office of Rare Diseases Research (ORDR)
OH
Ohio
Oliver S. Eng
Olympian
Omnibus
omnibus spending bill
omphalocele
On Target Laboratories Inc.
oncologist
oncology
Oncology Center of Excellence
OND
online educational tool
online support group
OOPD
op-ed
Open Act
Open Enrollment
Opposition Letter
Oprah Winfrey
Opsoclonus-Myoclonus Syndrome
optic atrophy
or Eosinophilic Esophagitis
Oregon
Organic Acidemia Association
orphan
orphan disease
Orphan Disease Update
Orphan Disease Update and NORD Online newsletter
Orphan Druct Act
orphan drug
Orphan Drug Act
Orphan Drug Act of 1983
Orphan Drug Act Resolution
orphan drug development
Orphan Drug Prices
Orphan Drug Pricing
orphan drug tax credit
orphan drugs
Orphan Drugs in the United States
Orphan Drugs in the United States: Rare Disease Innovation and Cost Trends Through 2019
orphan indications
Orphan products
Orphan Products Grants program
orphan therapies
Orrin Hatch
Oslo University Hospital
Osteogenesis Imperfecta Foundation
Otsuka America Pharmaceutical
Our Curse
Our Odyssey
out-of-pocket
Out-of-pocket costs
Oxbryta
Oxlumo
Pachyonychia Congenitia
PACS1-related syndrome
Pageant
PALS
Pamela Gavin
Pamela K. Gavin
pancreatic cancer
Pancreatic Cancer Action Network
pandemic
Panhematin
PAP
Parade
paraganglioma
parathyroid hormone
Parent
Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophyk Precision Medicine
Paroxysmal Nocturnal Hemoglobinuria
Partha Sen
partial trisomy 8Q duplication syndrome
Partner
Partners for Krabbe Research
Partnership
PassageBio
Passionate
Pat Furlong
Patent
PATH for PWS
patience assistance program
Patience Assistance Programs
Patient
Patient & Family Forum
Patient Access to Investigational Therapies
patient advocacy
Patient Advocate
Patient Advocate Awards
patient advocates
Patient Affairs Staff
Patient AirLift Services
Patient and Caregiver Resource Center
Patient and Family Forum
Patient and State Stability Fund
Patient Assistance
Patient Assistance Program
Patient Assistance Programs
patient care
Patient Community Building
patient data
Patient Engagement
patient engagement listening sessions
Patient Experience Data
Patient Focused
Patient Focused Drug Development Initiative
patient focused drug development meeting
patient journey
patient listening sessions
patient organizations
patient perspective
Patient Perspectives
Patient Powered
Patient Protections
patient registries
patient registry
patient registry data
Patient Service
patient services
patient stories
patient story
Patient Voice
patient-centered
patient-centered outcomes
Patient-Centered Outcomes Research Institute
patient-centric
Patient-First Blueprint
Patient-Focused Drug Development
patient-powered initiatives
Patient/Caregiver Speakers Bureau
patients
Patricia Richardson
Patrick Dunegan
Patrizia Cavazzoni
Patty Murray
Paul Frase
Paul Melmeyer
Paul Melmyer
Paul Newman
Paul Ryan
Paula Mann
pbm
PC Project
PCAST
PCD
PCD Foundation
PCI
PCOR
PCORI
PDSA
PDUFA
PEARLS Project
Pediatric
pediatric cancer
Pediatric ER
pediatric hypophosphatasia
Pediatric Minds Early Childhood Treatment Center
pediatric movement disorders
Pediatric Research Equity Act of 2003
Pediatrics
Pemazyre
Penn State University
Pennsylvania
Pennsylvania Rare Disease Advisory Council
People's Magazine
Perelman School of Medicine
peritoneal mesothelioma
personalized medicine
pet scan
Peter Frampton
Peter L. Saltonstall
Peter Marks
Peter Saltonstall
Petra Kaufmann
PFDD
PFIC
Pfizer
Ph.D.
Pharma
Pharmaceutical Medicine
pharmacies
pharmacy
pharmacy pediatrics club
pharmacy student
PharmaEssentia
PharmaEssentia Corporation
PharmaVOICE
PhD
Phenylketonuria
pheo
pheochromocytoma
PHI
Phil Maderia
Philip John (P.J.) Brooks
Phillip L. Pearl MD
PhRMA
physical therapy
Physician Education
Physician's Guide
Physicians
Pie Fight for PI
pigmented villonodular synovitis
pills
Pitt Hopkins Research Foundation
PKD
PKND
PKU
PKU Organization of Illinois
platelet
Platelet Disorder Support Association
PlatformQ Health
PlatformQ Health Education
PMP
PMP Research Foundation
PNH
poetry
POIS
Policy
Policy 101
Policy and Regulatory Affairs for NORD
Polycythemia Vera
Porphyria
Port Wine Stain Birthmarks
portacath
Portraits of Courage
Portraits of Courage Honoree
Portraits of Courage Honorees
position statement
Positive Exposure Ambassador
Post-Orgasmic Illness Syndrome
Post-Transplant Lymphoproliferative Disease
Post-Transplant Lymphoproliferative Disorder
Poster Abstracts
PPE
Prader-Willi
Prader-Willi Syndrome Association
Pravin Jadhav
Pre-Existing Condition Protections
pre-existing conditions
Pre-Existing Conditions Protections
pregnancy
pregnant
premium
premium assistance
premiums
prescription cost sharing requirements
Prescription Drug Cost Sharing
Prescription drug out-of-pocket cost protections
prescription drug spending
Prescription Drug User Fee Act
prescriptions
President Biden
President Donald Trump
President Obama
President Ronald Reagan
President Trump
Press
press release
pride month
Primary Distal Renal Tubular Acidosis
primary hyperoxaluria type 1
primary immunodeficiency
Primary Myelofibrosis
Primary Orhostatic Tremor
Primary Orthostatic Tremor
Principia Biopharma
Principles for Healthcare Coverage Reform
PRISMS
Proclamation
product review
Professor David Morris
Progeria
Project Baby Bear
project n95
Project RDAC
Promising Pathway Act
Protect Patients Now
protect your healthcare coverage
proteus syndrome
Providence Marathon
PRV Program
PSA
Pseudomyxoma Peritonei
Psychology Today
PTEN
PTEN hamartoma tumor syndrome
PTEN Hamartoma Tumor Syndrome Foundation
PTLD
public health
public health challenge
Public Health Leadership Award
Public Policy
Public Service Announcement
public-private partnership
pulmonary
pulmonary hypertension
Pulmonary Hypertension Association
Pulmonary Nontuberculous Mycobacterial Disease
pulmonary valve stenosis
pulmonology
pyknodysostosis
Pyruvate Kinase Deficiency
Q&A
Quincy M.E.
QuintilesIMS Institute
quotes
R&D
Rachel Sher
Rachid Tazi-Ahnini
Radium Hospital
Raise Awareness
Raise Your Hand Campaign
Raise Your Hand to Fight Rare Disease Research Campaign
Rally for Medical Research Hill Day
Ramaprasad Srinivasan MD
RAN
RAN State Ambassador
randi clites
Randy Rogers Band
Rapid Response Leadership Series
Rapid Response Seed Grant
Rare
Rare Access
RARE Act
Rare Action
Rare Action Netowrk
Rare Action Network
Rare Action Network State Ambassador
Rare Action Road Tour
Rare and Neglected Diseases Caucus
Rare and Undiagnosed Network (RUN)
Rare As One
Rare As One Project
rare autoimmune diseases
rare birth defect
rare blood disease
rare cancer
Rare Cancer awareness
rare cancer coalition
rare cancer day
Rare Cancer Day 2021
Rare Cancer Day 2022
Rare Cancer Foundation
rare cancer patient stories
rare cancers
Rare Caregiver Respite Program
rare disease
Rare Disease Advancemenet Research and Education Act
Rare Disease Advisory Council
Rare disease advisory councils
Rare Disease Advocacy
Rare Disease Advocates
Rare Disease Awareness
Rare Disease Clinical Research Network
Rare Disease Club
rare disease community
Rare Disease Congressional Caucus
Rare Disease Continuing Medical Education
rare disease cures
Rare Disease Cures Accelerator
Rare Disease Cures Accelerator Data and Analytics Platform
Rare Disease Database
Rare Disease Day
Rare Disease Day 2015
Rare Disease Day 2016
Rare Disease Day 2018
Rare Disease Day 2019
Rare Disease Day 2019. Rare Action Network
rare disease day 2022
Rare Disease Day 2023
Rare Disease Day U.S. Sponsor
Rare Disease Day US
Rare Disease Diversity Coalition
rare disease drug development
Rare Disease Education
Rare Disease Educational Support Program
rare disease facts
Rare Disease Funding Opportunities
Rare Disease Institute
Rare Disease Institute at Children's National Health System
rare disease nonprofits
Rare Disease of the Day
Rare Disease of the Week
rare disease patient
Rare Disease Patient & Caregiver Resource Center
rare disease patients
Rare Disease Pediatric Review Voucher Program
Rare Disease Report
Rare Disease Reports
Rare disease research
Rare Disease Research Grant
Rare Disease Research Grant Program
Rare Disease Respite
Rare Disease Summit
rare disease technology
rare disease therapies
rare disease treatments
Rare Disease Video Library
Rare Disease Videos
Rare Diseases
Rare Diseases & Orphan Products Breakthrough Summit
Rare Diseases Act of 2001
Rare Diseases Act of 2002
Rare Diseases and Orphan Products
Rare Diseases and Orphan Products Breakthrough Summit
Rare Diseases and Orphan Products Brekathrough Summit
Rare Diseases Clinical Research Network
Rare Diseases Clinical Research Network (RDCRN)
Rare Diseases Europe
Rare Diseases International
Rare Diseases Report: Cancers
Rare Diseases Report: Rheumatology
Rare Disesase Day
rare disorder
Rare Disorder Survey
Rare Disorder Toolkit
rare disorders
rare genetic cancer syndrome
rare genetic disease
Rare Impact
Rare Impact 2017
Rare Impact Award
Rare Impact Award Honoree
Rare Impact Award Honorees
Rare Impact Award Nominations
Rare Impact Awards
Rare Impact Awards Ceremony
rare lysosomal storage disorder
Rare Mom
Rare Moms
rare neurological condition
Rare Neurological Disease Special Report
rare neuromuscular and neurodegenerative diseases
Rare Pediatric Disease Priority Review Voucher
Rare Pediatric Disease Priority Review Voucher Program
Rare Revolution Magazine
Rare Spotlight
Rare Summit
RareAction
RareAction Network
RareCancer
RareCare
RareCare Patient Assistance
rareconnect
rareedu
RareInsights
RareLaunch
Ray Stevens
RCD 22
RCD'21
RDAC
RDACs
rdca
RDCA-DAP
RDCRN
RDD
RDD19 Story
RDDC
RDDNIH
RDEB
RDI
Reactive Arthritis
rebates
Rebecca Aune
Rebecca Campbell
Rebekah Bressi
Recap
Recessive Dystrophic Epidermolysis Bullosa
Recommended Uniform Screening Panel
Reconciliation
Reconstructive Surgery Act of 2005
Recurrent Respiratory Papillomatosis
Red Cross
Reed's Syndrome
Reflex Sympathetic Dystrophy
Regeneron
Regis College
Registered Nurse
registries
Registry
registry model
registry program
Regsitry
regulatory affairs
regulatory agencies
relief program
Remember the Girls
Renal Cell Cancer
Rep. DeGette
Rep. Upton
Report Card
Representative Bilirakis
Representative Butterfield
Representative Degette
Representative Henry Waxman
Representative Lance
Representative Margaret Craven
Representative Upton
Request for Proposal
Request for Proposals
Requests for Proposals
research
Research Director
Research Fund
Research Funding
research grant
Research Grants
Research Ready
research study
Research! America
ResearchMatch
resource
resources
Respite Care
Retaining Access and Restoring Exclusivity Act
Retevmo
Rethymic
Retinal Vasculopathy with Cerebral Leukodystrophy
Retinitis Pigmentosa
Retrophin
Rett Syndrome
Revised Senate Bill
RFP
RFPs
rheumatology
RIA
Rice University
Ricki Lewis
Ridgeback Biotherapeutics
Right to Try
Right to Try Act
Robert Califf
Robert Chen
Robert Long
Robert Lutz
Robert Rosenbloom
Rochester Institute of Technology
Rochester Minnesota
Rock and Roll Hall of Fame
RocketPharma
Ronald Reagan
Roscoe Brady
Rowan
Rozlytrek
Ruling
RUN
Running
Running for Rare
Running for Rare Diseases
Running for Rare Program
Running on Air
Running4Rare
Rush University Hospital
Rush University Medical Center
RUSP
Russ Illes
RVCL
Rx
Ryplazim
RYR-1 Foundation
RYR-1 gene
RYR-1 Related Diseases
S 4348
S. 201
S. 204
S.1878
safe step act
Samantha Scheer
Sanfilippo Syndrome
Sanfillipo Syndrome
Sanika Rane
Sanofi Genzyme
Sara's Cure
Sarah A. Taylor
Sarcoidosis
sarcoma
Savannah Hollis
Save Orphan Drugs
Saving Eliza
SB 7
scd
SCDAA
schools
Schuurs-Hoeijmakers Syndrome
scleroderma
Scleroderma Research Foundation
SCN2A
SCN2A Disorder
SCN8A Survey
SCOTUS
Sean Hepburn Ferrer
Seattle Children's Hospital
Secretary Azar
Secretary of the Department of Health and Human Services
Section 1115 waivers
SELENON/SEPN1-related myopathy
Senate
senate briefing
Senate Cures
Senate Cures Legislation
Senate Finance Committee
Senate Health Education Labor and Pensions Committee
Senate Healthcare Bill
Senate Innovations for Healthier Americans Initiative
Senator
Senator Bill Cassidy
Senator Brown
Senator Edward Kennedy
Senator Elizabeth Warren
Senator from Washington
Senator Hatch
Senator Michael Bennet
Senator Nancy Kassebaum
Senator Orrin
Senator Orrin Hatch
Senator Richard Blumenthal
Senator Richard Burr
Senator Roberts
Senator Tammy Baldwin
Senator Ted Kennedy
September 11th
September 30
Sequestration
Seth Rotberg
Severe Combined Immune Deficiency (SCID-ADA)
severe weather
SGS
Shafali Spurling Jeste
Shaquille O'Neal
Share Your Story
Sheba Medical Center
Shel Silverstein
Sheldon Schuster
sherrod brown
Shine Light on Rare Diseases
Shire
Short Bowel Syndrome
Shortage
Show Your Rare
Show Your Stripes
Show Your Stripes Challenge
Shprintzen Goldberg Syndrome
sickle cell disease
Sickle Cell Disease Association of America
SIDS
Sign up today
Sika Dunyoh
Silicon Valley Community Foundation
Silver Sponsor
Simran Handa
Sing me a song
Sitosterolemia
Sitosterolemia Foundation
skill
Skin Disorders
SLC6A1
SLC6A1 Connect
Sleep
Sly syndrome
SMA
SMS
snowstorms
Snyder-Robinson Syndrome
social distancing
social media
Social Security Administration
social support
software platforms
Sophia Hanson
South Carolina
space center houston
spacticity
Special Books by Special Kids
special education
special enrollment periods
Special Moments
Special Olympics
specialty drug spending
Spectrum vs. Burwell
spencer health solutions
Spending Bill
Spinal Muscular Atrophy
Spinocerebellar Ataxia
Spinocerebellar ataxia autosomal recessive 9 (SCAR9)
Sponsor
Sprengell Deformity
SPS
SRS
ssa
SSI
St. Baldrick’s Foundation
standards of care
Stanford University
STAR Act
STAT
state
State Advocacy
State Ambassador
state capitol
state government
state governments
state health departments
state house
State House Event
State House Events
state issues
State Legislation Tracker
State Policy
State Policy Legislative Tracker
State Progress Report
State Report Card
State Report Carfd
State Representative from Massachusetts
state-by-state
state-regulated health insurance
Stealth BioTherapeutics
stem cell transplant
step therapy
Stephanie Cindric
Stephen Cederbaum
Stephen Friedhoff
Stephen Groft
Stephen Hahn
Steven Grossman
stiff body syndrome
Stiff Person Syndrome Research Foundation
stiff persons syndrome
stitches
Stripe Out Your School
stroke
Strong
Strongbridge
Stronger Scholarship
student leaders
Student Membership
Students
Students for Rare
Students for Rare Chapter
study
stupid cancer
stupid cancer show
Sturge Weber Foundation
Sturge-Weber Syndrome
STXBP1
STXBP1 encephalopathy
STXBP1 Foundation
STXBP1 Move to Cure
sub-study
SUDC
Sudden Infant Death Syndrome
Sudden Unexpected Death Data Enhancement and Awareness Act (H.R. 669)
sudden unexplained death in childhood
Suddenly Sleepy Saturday
Sudhir Borgonha
Summer Camp
Summit
SUOX mutation
Supplemental Security Income
Support
Supreme Court
surprise medical bills
Survey
survey findings
Susan A. Berry
Susan Fernback
Susan G. Komen
Sylvia Burwell
symptoms
Syringomyelia
systemic scleroderma
SZB
Tai Spargo
take action
Takeda
TANGO2 disorder
TANGO2 Research Foundation
Tara Notrica
target cancer foundation
TargetCancer Foundation
Tatton Brown Rahman Syndrome
Tatton Brown Rahman Syndrome Community
tax credit
Tax Cut and Jobs Act
Tax Cuts and Jobs Act
tax reform
Taylor Kane
TBRS
TBRS Community
TCS
TCS New York City Marathon
Teachers
TEDx
telehealth
Telehealth Extension and Evaluation Act
telehealth principles
telehealth services
telemedicine
Telomore
Tepezza
Terry Jo Bichell PhD
Texas
Texas Children's Hospital
Texas Rare Action Network
Texas Rare Action Network State Ambassador
Texas State Ambassador
Thank You
Thanksgiving
The 100 Days Agenda: A Patient-First Blueprint
The Adrenoleukodystrophy (ALD) Foundation
The ALS Association
The Association for Creatine Deficiencies
The Atypical HUS Foundation
The Avalon Foundation
The Awesome Disease
The Balancing Act
The Calliope Joy Foundation
The Children’s Hospital of Philadelphia
The Chondrosarcoma Foundation
The Critical Path Institute
The Cute Syndrome Foundation
The David Ashwell Foundation
The David Bach Consort
The Dup15qAlliance
The Galactosemia Foundation
The Guthy-Jackson Charitable Foundation
The Hill
The Hole in the Wall Gang Camp
The Honorable Michael McCaul
The Honorable Michael McCual
The Honorable Sarah Davis
The Hope Fund
The Legacy of Angels Foundation
The Marfan Foundation
The Mighty
The Moebius Syndrome Foundation
The Morgan Leary Vaughan Fund
The Motley Fool
The NORD Guide to Rare Disorders
The OMSLife Foundation
The Physician’s Guide
The Power of Patients
The Power of Patients: Informing Our Understanding of Rare Diseases
The Rare Disease Cures Accelerator-Data and Analytics Platform
The Recessionals
The Snow Foundation
The Snyder-Robinson Syndrome Foundation
The United Mitochondrial Disease Foundation
The University of Chicago
The University of Sheffield
The VEDS Movement
The Week
TheMighty.com
therapeutic recreation
Therapeutics for Rare and Neglected Diseases (TRND)
therapy
Theresa Mullin
Theresa Mullin PhD
ThinkGenetic
Thoma Family
Thomas Jefferson
Thomas M. Haberman
tik tok
Tim Boyd
Timothy Yu
titanium rib
TK2 Mitochondrial DNA Depletion Syndrome
Together We Are Strong
Tom Rath
tom rhoads
Tom Rusin
Tony Ferrandino
toolkit
Tourette Syndrome Foundation
toxic acute progressive leukoencephalopathy
Toxic Shock Syndrome
tracheobronchomalacia
Travere Therapeutics
Travis Flores
treatment
treatment protocols
Trevor Noah
triaging
TRICARE
Trikafta
Trio Health
Tristan Lee
Trump Administration
TSC New York City Marathon
TSS
TUBB4A gene mutation
tuberous sclerosis complex
tumor
tumors
Turalio
Turner Syndrome
Tweetchat
twins
Twitter
U.S. Court of Appeals
U.S. Food and Drug Administration
U.S. House of Representatives
U.S. Olympian
U.S. Senate
UCLA
UDN
UFA
Ulcerative Colitis
Ultragenyx Pharmaceutical Inc
UMass Memorial Hospital
UMDF
underrepresented populations
Undiagnosed
Undiagnosed Day
Undiagnosed Diseases
Undiagnosed Diseases Network
Undiagnosed Diseases Patient Assistance Program
Undiagnosed Diseases Program (UPD)
undiagnosed patients
Undiagnosed Patients Registry
undiagnosed registry
United Healthcare
United Leukodystrophy Foundation
United Mitochondrial Disease Foundation
United States
United States Senate
United Therapeutics Corporation
University Health Network
University Hospital of Pisa
University of California
University of California Los Angeles
University of California San Francisco
University of Manitoba
University of Maryland
University of Maryland School of Pharmacy
University of Michigan
University of Milan
University of Minnesota
University of Notre Dame
University of Pennsylvania
University of Wyoming
University of Zurich
unmet need
Urea Cycle Disorders
US
US Congress
US Department of Health and Human Services
US Food and Drug Administration
US Food and Drug Administration Commissioner
US Hereditary Angiodema Association
US News and World Report
user fee
user fee package
user fee reauthorization
user fees
Utah
uveal melanoma
vaccination prioritization
vaccine
vaccines
Vanda
Vanderbilt Kennedy Center for Excellence in Developmental Disabilities
Vanderbilt University
Vanessa Boulanger
Vanessa Devore
Vascular Ehlers-Danlos Syndrome
Vasculitis
VCP
Venture Philanthropy
VEPTR
Vermont
Vertex Pharmaceuticals
Vertical Expandable Prosthetic Titanium Rib
veto
VHL
VHL Alliance
Vice President
Vice President of Regulatory and Government Affairs
video
video submissions
videos
Violins for Vasculitis
viral videos
Virginia
virtual event
Virtual Hill Day
virtual platforms
Virtual Race
virtual tools
Voice of America
voice of rare disease
Voice of the Patient
Voice of the Patient Report
Voices of Rare Cancer
volunteer
von Hippel-Lindau
Waldenstrom Macroglobulnemia
Walker-Warburg syndrome
Warner Theatre
Washington D.C.
Washington DC
Washington Post
Washington State University
Washington University School of Medicine
Ways and Means
Webinar
WebMD
WEGO Health
Wellstat Therapeutics Corporation
Wendy Chung MD PhD
Westminster College
White House
white paper
whole exome sequencing
whole genome sequencing
Wilbur Bowne
Wilhelm Foundation
will
William & George Akers Private Foundation
William Akers
William Cruz
William Yank
Williams Syndrome
Wipro
Wiskott-Aldrich Syndrome
WJLA-TV
Wolfram Syndrome
workshop
world blood donor day
World Cholangiocarcinoma Day
World Health Day
World Orphan Drug Congress
worldwide collaboration
Worldwide Syringomyelia & Chiari Task Force
Worldwise Syringomyelia & Chiari Task Force
Worth magazine
WSCTF
WWS
www.rarediseases.org
X International Pemphigus & Pemphigoid Foundation
X-linked diseases
X-linked myotubular myopathy
Xavier Becerra
xeroderma pigmentosum
XLH Network Inc.
Yair Ankster
Yann Le Cam
young adult rare disease
Young Investigator Grant
Zafgen
Zavesca
zebra
Zika
Zokinvy
Zolgensma
zollinger-ellison syndrome
Set Valu
Keyword Search
Categories
Select an option
#NORDintheNews
Advocacy
Advocacy>Press Releases
COVID-19
Events
Featured News
Get Involved
Head of the Herd
Head of the Herd
Industry
Medicaid
Medical
Medical Affairs
Members
Newborn Screening
NORDPod
Partnerships
Partnerships>Rare Cancer Coalition
Partnerships>RDCA-DAP
Patient Stories
Patient Stories>Voices of Rare Cancer
Press Releases
Press Releases
Rare Cancer Coalition
Rare Disease Day
RareInsights
RareLaunch
RDACs
RDCA-DAP
RDCA-DAP
Registries
Research
Research>Registries
State Report Card
Sticky Posts for Homepage
Students for Rare
Uncategorized
Voices of Rare Cancer
Voices of Rare Cancer 2020
Tags
Select an option
{eter L. Saltonstall
#1in10
#1in10 Campaign
#BLM
#DoYourShare
#LightUpForRare
#LivingRareForum
#MissAmericaVote
#NORDGala15
#NORDinthenews
#NORDReport
#NORDSummit
#ProtectPatientsNow
#RareCancerDay
#RareDiseaseDay
#RareDiseases
#RareERChat
#RareImpact
#RDD18Chat
#SaveCHIP
#SaveOrphanDrugs
#ShowYourRare
#ShowYourStripes
#SSANDFORUM
#TCSNYCMarathon
000 Mile Rare Movement
1 in 10
100 Days Agenda
1983 Orphan Drug Act
2015
2016
2018 Rare Impact Award Honoree
2018 Rare Summit
2018 State Report Card
2019
2019 NORD Rare Summit
2019 Official Rare Disease Day Video
2019 Rare Impact Award Honoree
2020 Living Rare
2021
2022 NORD Rare Diseases and Orphan Products Breakthrough Summit
2022 Rare Disease and Orphan Products Breakthrough Summit
21st Centure Cures
21st Century Cures
21st Century Cures Act
22q11.2 deletion syndrome
2q23.1 Microdeletion/Duplication
2seventy bio
3-D printing
30 million
35 ways and growing
35 Years and Growing
35th Anniversary
35th Anniversary Celebration
35th Anniversary Celebration presenting the Rare Impact Awards
5 Myths
50 states
501(c)(3)
5K
7
7000 Mile Rare Movement
A Cure for Ellie
A Cure in Sight
A Short Walk Home
AA amyloidosis
AAC device
AAMDS
AAMDS International Foundation
AAMDSIF
Aaron Hamvas
aarskog syndrome
Abbey Meyers
Abbey S. Meyers
Abbey S. Meyers Leadership Award
Abby Norman
ABC News
ABCG5
abcg8
Abecma
Abeona Therapeutics
Abetalipoproteinemia
Abortion
ACA
ACA Individual Mandate
Academy of Country Music
Academy of Country Music Lifting Lives Music Camp for People with Williams Syndrome
accelerated approval
accelerated approval pathways
Accelerated Approval Program
Access
access to treatments
accessibility
ACD
ACD/MPV
ACDMPV
ACIP
ACIS
ACPMP
ACPMP Research Foundation
Acting Commissioner
Action Potential Grant
Acute lymphocytic leukemia
acute myeloid leukemia
acute myeloid lukemia
ADA
Adam Rose
Adaptive Marketing
adaptive yoga
adherence
adrenoleukodystrophy
Adriano Aguzzi
Adult Polyglucosan Body Disease (APBD) Research
Adult Rare Disease Patient
Advanced Premium Tax Credits
Advances in Pulmonary Hypertension
advancing drug development
Advancing Hope Act
Advancing Hope Act of 2016
Advancing Hope Act of 2016 (S.1878)
Advancing Innovation
Advancing Research for Neurological Diseases Act (S. 849)
Advancing Targeted Drugs for Rare Diseases Act
Advancing Targeted Therapies for Rare Disease Act
Advisory Committee on Immunization Practices
advocacy
advocacy alert
Advocacy Events
advocacy organizations
Advocate
affordability
Affordable Care Act
Affordable Care Act marketplace
afibrinogenemia
Agency for Healthcare Research and Quality
Agios
AGMD
AHCA
AHDS
aHUS
AI
AIDS Institute
Aileen Tobin
Aimee Guarnieri
AKU Society
ALD
Alex Azar
Alexa Moore
Alexander Pham
Alexandra Freitas
Alexion
Alexion Pharmaceuticals
ALIBER
Alison and Paul Frase
Alison Rocket Frase
Alison Rockett Frase
Aliza
Aliza Fink
ALL
All Things Kabuki
Allan-Herndon-Dudley syndrome
Alliance for a Stronger FDA
Alliance for Aging Research
Alliance for Connected Care
Allison Lint
alnylam pharmaceuticals
ALPS
ALS
ALS Association
Alston & Bird
alveolar capillary dysplasia
Alveolar Capillary Dysplasia Association
Alveolar Capillary Dysplasia with Misalignment of the Pulmonary Veins
Alynylam Pharmaceuticals
Ambassador
Ambry Genetics
AMENSupport
American Academy of Pediatrics
American Behcet's Disease Association
American Cancer Society
American Cancer Society Action Network
American Cancer Society Cancer Action Network
American College of Cardiology
American College of Medical Genetics and Genomics
American Health Care Act
American Healthcare Act
American Heart Association
American Lung Association
American Medical Student Association
American Medical Students Association
American Multiple Endocrine Neoplasia
American Multiple Endocrine Neoplasia (AMEN) Support group
American Patients First
American Plasma Users Coalition (A-PLUS)
American Porphyria Foundation
American Public Health Association
American Society of Hematology Annual Meeting
American University of Integrative Sciences School of Medicine
Americans with Disabilities Act
Amgen
amicus brief
Amicus Therapeutics
amniotic band syndrome
AMSA
Amyloidosis
Amyloidosis Support Groups Inc.
amyotrophic lateral sclerosis
Anagralide
and The Physician’s Guide series of booklets
and Treatment in the US: A 30-year Comparative Analysis
Andrew W. Mellon Auditorium
Angelia Benson
Angelman Syndrome
Ann & Robert H. Lurie Children’s Hospital of Chicago
annual report
Annual Report 2021
Anthem
Anthem Foundation
anthrax
anti-discrimination
anxiety
Apellis
Apellis Pharmaceuticals Inc
APF
Aplastic Anemia and MDS International Foundation
appendix
Appendix Cancer
Appendix Cancer and Pseudomyxoma Peritonei
Appendix Cancer/Pseudomyxoma Peritonei Research Foundation
approval standards
APS Type 1
APS Type 1 Foundation
APS-1
arachnoiditis
arginase deficiency
Arlington
Armed Services Committee
Art Alexakis
art exhibit
Arthritis Foundation
arthrogryposis
Arthur Caplan
ASGCT
ASH
Asklepion Pharmaceuticals
Assembly Education Committee
Associate Director for Strategic Initiatives
Association for Accessible Medicines
Association of Gastrointestinal Motility Disorders Inc.
Astellas Pharma
asthma and allergy foundation of america
AstraZeneca
Atara
ataxia
Athlete
atrial septal defect
Atypical HUS
Audrey Hepburn
August
autoimmune disease
Autoimmune Lymphoprolifertive Syndrome
autoimmune neutropenia
Autoimmune Polyglandular Syndrome Type 1
autonomic dysfunction
autosomal genetic disorder
Avalere
Avalere Health
AveXis
award
award nominations
Awards Ceremony
Aware of Angels
awareness
awareness day
Ayvakit
Azar
Baby Formula
Baby's First Test
Bailey Gribben
balance bill
BAM Syndrome
Banner on the Moon
Barb Calhoun
Barbara Hollister
barriers
Barriers to Rare Disease Diagnosis
Batten Disease
BBB
BBBA
BCRA
BDSRA
Be a Hero
Beau Biden
Beckwith-Weidemann Syndrome
Behcet's disease
Behind the Mystery
Believe Limited
bequest
Bespoke Gene Therapy Consortium
Besremi
Best Pharmaceuticals for Children program
beta-thalassemia
Beth Nguyen
Better Care Reconciliation Act
Better Care Reconciliation Act of 2017
Beverley Francis-Gibson
BGTC
bicycle
Bilary Atresia
bile duct cancer
biliary atresia
Bill
Bill Erickson
Billy Dunn
BIO
biobank
bioengineering
Biogen
biological pathways
biologics
BioMarin
BioMarin Pharmaceutical
BioMarin Pharmaceutical Inc
BioNews Service
BioNJ
BioNTech
bioprinting
biosimilars
Biotechnology Industry Organization
bioterrorism
birth defect
Black Bone Disease
black lives matter
Black Women’s Health Imperative
Block Grants
Blood
blood cancer
blood cancer awareness month
blood counts
Blood Disorders
bluebird bio
Blueprint
Blueprint Medicines
BoardSource
Bob Saget
Boehringer Ingelheim
Boler-Parseghian Center for Rare and Neglected Diseases
bone cancer
bone marrow
bone marrow transplant
Bonnell Foundation
Bonnie Royster
book
Book of Hope
Bosma arhinia microphthalmia (BAM) syndrome
Boston Children's Hospital
Boston Marathon
Boston Marathon 2015
Boston Medical Center
brain
Brain Cancer
Brain Tumor
Brandon Hudgins
Brandstar
Breakthrough Summit
Breakthrough Therapy Designation
breast cancer
Brent Clough
Brian Kemp
Bridge the Gap-SYNGAP Education and Research Foundation
BridgeBio Pharma
Bristol Myers Squibb
Brugge Breast Cancer Lab
Budapest
Build Back Better
Build Back Better Act
bullying
Bustle
C-Path
CA
Cabergoline
CACNA1A
California
California Attorney General Xavier Becerra
Call Center
Call for Posters
Calliope Joy Foundation
Cancer
Cancer in our Genes Patient Databank
Cancer Moonshot
Cancer Support Community
cannabinoids
capacity-building
Capitol Hill
CARD9 Deficiency
cardiac
cardiology
cardiomyopathy
Care
caregiver
Caregiver Respite
caregivers
Caring for Carcinoid Foundation
Carmina Taylor
Carolyn Sheridan
CAT
Cat Eye Syndrome
CAT Scan
Catalyst Pharms.
catecholamines
Catherine Blansfield
Cathleen Lutz
Cavernous Angioma
CBER
CBO
CBS News
CCF
CCHS
CCHS Family Network
CCHS Foundation
CCPA
CCS
CDC
CDER
CDER’s Associate Director for Strategic Initiatives
CdLS
CdLS Foundation
CDRH
celebration
Celera Genomics
Center
Center for Biologics Evaluation and Research
Center for Drug Evaluation and Research
Center for Drug Evaluation and Research (CDER)
Center for Innovation in Global Health
Center for Responsible Science
Center of Excellence
Centers for Disease Control
Centers for Disease Control and Prevention
Centers for Medicare and Medicaid Services
centers of excellence
central congenital hypoventilation syndrome
centronuclear
cerebral palsy
Certified Nursing Assistant
Chagas
Chalk the Walk
CHAMP1
Chan Zuckerberg Initiative
Chandra Clark
Change Together
Charity Navigator
Charity of the Week
Charity Partner
charity running team
Charlene Son Rigby
Charlene York
Chasten Buttigieg
chemo
chemo brain
chemotherapy
CHI
Chiari Malformation
Chicago
Chicago Illinois
chief medical and scientific officer
childhood cancer
Children's Cardiomyopathy Awareness Month
Children's Cardiomyopathy Foundation
Children's Health Insurance Program
Children's Hospital of Los Angeles
Children's Hospital of Philadelophia
Children's Hospital of Philadelphia
Children's Inn
Children's National
Children's National Director
Children's National Health System
Children's National Hospital
Children's National Medical Center
Children’s Cardioymyopathy Foundation
Children’s Connecticut Medical Center
Children’s Hospital and Hackensack Meridian School of Medicine
Children’s Miracle Network
CHIP
CHOC Children’s
CHOC Children’s Hospital
cholangiocarcinoma
Cholangiocarcinoma Foundation
Chondrosarcoma
CHOP
CHOP Leukodystrophy Center
chris anselmo
Chris Brannigan
Chris Ulmer
Christine Grube
Christopher Anselmo
chromosome 16
chronic disease
Chronic Disease Coalition
chronic Epstein-Barr virus (EBV)
Chronic Granulomatous Disease
Chronic Illness
chronic inflammatory demyelinating polyneuropathy
chronic migraine
chronic pain
Chung Lab
Cicatricial Alopecia Research Foundation (CARF)
CIDP
CIDP Foundation
CIDP Foundation International
Cincinnati Children's Hospital Medical Center
Cindy Abbott
Citizen Petition
Citizen's Petition
CL Foundation
Claude Steriade
clear cell sarcoma
Cleveland
Cleveland Clinic
Clinic for Special Children
clinical trials
CLOVES Syndrome
CLOVES Syndrome Community
CME
CMS
CMTC-OVM
CNA
CNM
CNTNAP1 mutation
CO
co-pays
Coalition
Coalition Statement
Coats Plus Syndrome
Cockayne Syndrome
CoE
Coffin-Siris Syndrome
collaboration
Colleen King
Colonel Ken Cameron
colorado
Columbia Presbyterian
Columbia University Irving Medical Center
Columbia University Medical Center
Columbia University’s Fu Foundation School of Engineering
COMBINEDBrain
Commissioner
Commissioner of the FDA
community
community survey
Community Survey Report
compassionate allowances
Compassionate Use
compliance
computer
computer-assisted diagnosis
Conduct of Clinical Trials of Medical Products during COVID-19 Public Health Emergency
conference committee
congenital central hypoventilation syndrome
congenital heart defects
congenital hyperinsulinism
Congenital Hyperinsulinism International
congenital muscular disorder
congenital muscular dystrophy
Congress
Congressional Budget Office
Congressional Childhod Cancer Congress
Congressional Leadership
Congresswoman Doris Matsui
Congresswoman McMorris Rodgers
Congresswoman Robin Kelly
Connecticut
Connecticut magazine
Connecticut-based nonprofits
Connecting ALS
Constitution
Consumer Assistance Program
continuing medical education
Continuing Resolution
contracture
COPA Syndrome Foundation
copay accumulator programs
copay accumulators
COPPA
CORD
Cornelia de Lange Syndrome
Cornelia de Lange Syndrome Foundation
coronavirus
cost of orphan drugs
cost-sharing
Council of National Alliances
Court
Court Case
coverage
covid
covid-19
COVID-19 Premium & Medical Relief Program
COVID-19 Rapid Response Leadership Series
covid19
CR
Craine’s Cholangiocarcinoma Crew
Creating Hope Act
Creutzfeldt-Jakob Disease
crisis communications
crisis management
CRISPR
Cristina Y. Go MD
Critical Path
Critical Path Institute
CRMCC
CRN Family Conference
Crohn’s disease
Crowdfunding
CSS
CTCL/MF
CTF
Cure CMD
Cure MLD
Cure SMA
CureDuchenne
CureDuchenne Cares
CurePSP
CuresNow
Curing Retinal Blindness Foundation
Cushing's Disease
cutaneous lymphoma
Cutaneous Lymphoma Foundation
cutaneous T-cell
Cutis Laxa
Cutis Laxa Internationale
cutis marmorata telangiectatica congenita
cyclodextrin
Cystic Fibrosis
Cystic Fibrosis Foundation
Cystic Fibrosis Research Inc.
Cystinosis
Cystinosis Research Network
Cytalux
CZI
Dad
Daiichi-Sankyo
Dana-Farber Cancer Institute
Daniel Stanley
Daniela Delgado
Daniele Batlle
Danny's Dose
Danyelle Sun
Data
Data-Sharing
DataRevive LLC
Davelyn Hood
David Arons
David Cry
David Geffen School of Medicine at UCLA
David Lebwohl MD
David Meeker
Dawn Laney
day of action
Days of Action to Protect Your Heatlhcare Coverage
DC
deafness
Deanna Portero
Debbie Drell
Debbie Skolaski
Deborah Skolaski
DEBRA International
Debra Miller
Debra of America
Debra Regier MD PhD
DEI
Delaware
Dennis Jackman
Department of Health & Human Services
Department of Health and Human Services
Department of Insurance
dermatology
Desiree Lyon
Desmoid Tumor Research Foundation
development
developmental delay
Devic syndrome
Devin Alvarez
DHHS
DHPS
DHPS Foundation
DHPS Patient Registry
diabetes insipidus
diabetes mellitus
Diagnosis
diagnostic odyssey
Diagnostic Testing
diagnostician
diagnostics
Diane Dorman
diaphoresis
Diazoxide
DiGeorge syndrome
Digestive Motility Disorders
digital health
DIPG
direct-to-consumer genetic testing
Director of Marketing and Communications
Director of Membership
Director of Patient Services
Director of Research
Director of the Center for Drug Evaluation and Research at the Food and Drug Administration
Director of the Center for Thoracic Insufficiency Syndrome
Director Walensky
Disabilities
disaster relief for rare disease patients
disease
disease-specific Emergency Relief Programs
Disney
distal 18q deletion
Diversity
Division Chief of Genetics and Metabolism
Division of Endocrinology at Northwestern
DMD
DNA
DNMT1 gene
DNMT3A
Dobbs v Jackson
Doctorate of Nursing Practice
doctors
documentary
Dolly the sheep
Dompe
Donate
Donate to NORD
donation
Donna Appell
Donna Appell RN
Download
Dr. Amanda Cohn
Dr. Anita Gupta
Dr. Christoper Austin
Dr. Christopher Austin
Dr. Christopher Klein
Dr. Cynthia Tifft
Dr. Diego Fornasari
Dr. Donald C. Vinh
Dr. Eli Diamond
Dr. Francis S. Collins
Dr. Frederick Kaplan
Dr. Janet Woodcock
Dr. John Graham
Dr. Juvianee Estrada-Veras
Dr. Katherine Leaming
Dr. Levy
Dr. M Kathryn Liszewski
Dr. Margaret Hamburg
Dr. Marshall Summar
Dr. Marshall Summars
Dr. Marty Makary
Dr. Melinda Burnworth
Dr. Michael F. Goldberg
Dr. Michelangelo Mancuso
Dr. Michio Hirano
Dr. Natalie Shaw
Dr. Noonan
Dr. Pantea Sharifi Hannauer
Dr. Peter Marks
Dr. Rachel Harper
Dr. Richard Besser
Dr. Richard Pazdur
Dr. Robert Califf
Dr. Robert Campbell
Dr. Robert Coleman
Dr. Robert M. Campbell
Dr. Scott Gottlieb
Dr. Stephen Hahn
Dr. Stephen Ostroff
Dr. W. French Anderson
Dr. Wendy Chung
Dr. Yeboa
draft guidance
Dravet and Hurler syndromes
Dravet Syndrome
Dravet Syndrome Foundation
Drexel University
drug costs
drug development
drug development process
drug discovery
Drug Manufacturers
Drug Prices
drug pricing
drug research
drug sales
dTRA
duchenne muscular dystrophy
Duct Tape Won't Fix This
Duke University
Dynata
dysarthria
dyslexia
Eallonardo Family
early diagnosis
Ebanga
EBRP
EBV
ECD Global Alliance
ECD Patient and Family Gathering
ECD Referral Care Centers
ECMO
Ectodermal dysplasia
Ed Silverman
Editas Medicine
Editorial Collaboration
EDS
Eduational Initiatives
Education
Educational Event
Educational Initiatives
educational video
Educational Webinar
edward neilan
Ehlers Danlos Syndrome
Ehlers-Danios Syndrome
Ehlers-Danlos
Eiger Biopharmaceuticals
EL-PFDD
Eleanor Perfetto
Eli Lilly
Elisabeth Dykens
Eliza Rista
Elizabeth Katz
Elizabeth Silva Anderson
Ellen V. Sigal
Ellie McGinn
Emergency Relief Resources Packet
Emergency Rom Visits
Emergency Room Protocol
emergency use authorization
Emily Argersinger
Emory University
Emory University Department of Human Genetics
Empaveli
End NF
endocrinology
Energy and Commerce Subcommittee
Energy for Life Walkathon
Ensuring Access to Clinical Trials Act
Entering a New Era
Enzyvant Therapeutics Inc
epidermolysis bullosa
Epilepsy Foundation
epileptic encephalopathies
Epstein-Barr virus
equality
equity
ER with a Rare Disease
Erdheim Chester Disease Awareness Week
Erdheim-Chester Disease
Erica Barnes
Ernst & Young
essential health benefits
Essential Thrombocythemia
estate
estate plan
Estelle Benson
ET
ethics
EUA
Eugene Washington Engagement Award
EURODIS
European Commission
European Union
eurordis
EURORDIS-Rare Diseases Europe
Eva Juneja Foundation
Eva's Butterfly Wishes for Walker-Warburg Families
Event
Events
Eversana
Eversource Hartford Marathon
EveryLife Foundation for Rare Diseases
Expanded Access
externally led Patient-Focused Drug Development
externally-led Patient-Focused Drug Development Meeting
extracorporeal membrane oxygenation
EY
Eye Believe Patient Survivorship Meeting
Fabry Disease
Facebook
Facebook Live
factor-vii deficiency
Familial Hypercholesterolemia
Families
FamilieSCN2A Foundation
family
Fanconi Anemia
Fanconi Anemia Research Fund
FAQ
FARF Translational Science Director
Father
Father's Day
FD/MAS Alliance
FDA
FDA Acting Director
FDA CDER
FDA Commissioner
FDA Commissioner Stephen Hahn
FDA Matters
FDA OCE
FDA Oncology Center of Excellence
FDA Patient Affairs
FDA Public Meeting
FDA Rare Diseases Program
FDA Reauthorization Act
FDA Reauthorization Act of 2017
FDA Safety & Innovation Act
FDA Safety and Innovation Act
FDA user fees
FDARA
FDASIA
Febrile Infection-Related Epilepsy Syndrome
February 28
February 28th
February 29
federal government
federal legislation
federally regulated insurance
fibrolamellar
Fibrolamellar Cancer
Fibrolamellar Cancer Foundation
fibrolamellar carcinoma
fibromyalgia
financial assistance
financial support
Financial Times
Finding a Zebra Among Many Horses
FIRES
FIRST
Florida
Food & Drug Administration
Food and Drug Administration
Food and Drug Administration Reauthorization Act of 2017
Food and Drug Amendments of 2022
FOP
Forming a Foundation
Formula
Formula Shortage
formulary
Foundation for Ichthyosis and Related Skin Types
Foundation for Prader-Wili Research
Foundation for Prader-Willi Research
Foundation for Rare Blood Diseases
Foundation to Fight H-abc
FOXF1
FOXF1 gene
FPIES Foundation
Frances Flanagan
Francis Collins
Frank Sasinowski
Fred Upton
Free Will
FreeWill
Friedreich's ataxia
Friedreich’s Ataxia Research Alliance
Friends of Cancer Research
From Emotions to Advocacy by Pam Wright and Pete Wright
Frontline Medical Communications
Full House
Fundraiser
fundraising
Gabriel Low
Gain Therapeutics
galactocerebrosidase
galactosemia
Galactosemia Foundation
GALC
GAN
GARD
Garrett Mann
gastroenterology
gastrointestinal stromal tumors
Gastroschisis
Gaucher Disease
GBS
GBS CIDP Foundation
GBT
GDPR
Gene
gene editing
gene mutation
gene replacement therapy
Gene therapy
Genentech
generics
genes
Genet Testing
Genetech
genetest101
genetic
Genetic and Rare Diseases Information Center
genetic biobanks
Genetic Counselor
genetic disease
genetic disorder
genetic testing
Genetics
genome editing
genomic testing
Genzyme
George Soros
Georgetown School of Medicine
Georgia
Get Involved
Get Invovled
gift
GIST
Give Kids the World
Giving Spotlight
Giving Spotlight. Giving to NORD
Giving to NORD
Givlaari
GlaxoSmithKline
Glenn S. Williams
Global
Global Blood Therapeutics
Global Chain of Lights
Global Foundation for Peroxisomal Disorders
Global PNH Patient Registry
Global Video
globoid cell leukodystrophy
Glybera
Good Governance
Good Morning Peyton
Gorlin Syndrome
Gorlin Syndrome Alliance
government
governor
Governor Jared Polis
Governor Kemp
Governor Matt Bevin
Governor of Kansas
governors
Grace Rista
Graham-Cassidy
Graham-Cassidy Bill
grant
grants
Guadalajara Mexico
Guest Blog
Guest Blog Post
guest blogger
Guillain-Barre syndrome
Guillan-Barré syndrome
Guthy-Jackson Foundation
GW Pharmaceutics
gymnastics
H-abc
H.R. 3783
H.R. 669
HAE
Hal Dietz M.D.
Hand Prints Across America
Handprints Across America
Hannah Kane
Hannah's Hope
Hao Cheng
Harjot Randhawa
Harvard Medical School
Harvard T.H. Chan School of Public Health
Hashimoto syndrome
HB239
Head of the Herd
Health
Health & Human Services
Health and Human Services
Health and Human Services Secretary
Health Coverage Reform
health crisis
health disparities
health disparity
health equity
health insurance
health insurance marketplaces
health programs
healthcare
Healthcare Bill
healthcare costs
Healthcare Coverage
healthcare reform
healthcare.gov
HealthDay
Healthline
Healthwell Foundation
Heart Disease
hEDS
Heidi Ross
HELP Committee
Helping Hands for GAND
hematology
hemophilia A
Hemophilia Federation of America
Henry Waxman
Hepatocellular carcinoma
hepatology
Hereditary Leiomyomatosism
Hereditary Neuropathy Foundation
Hereditary Sensory Autonomic Neuropathy Type 1E
Heriditary Angiodema
Hermansky Pudlak Syndrome
HFA
HHS
HHS Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs
HI
HI Global Registry
hidradenitis suppurativa
high-risk pool
Highlights Video
HIPAA
histiocytes
histiocytosis X
HLA B27
HLA B27 autoimmune disease
HLRCC Family Alliance
Home Health Aide
home infusions
HOME Study
HomeServe USA
Homocystinuria
Homocystinuria due to Cysathionine Beta-Synthase Deficiency
Hope for Hasti
Horizon
Horizon Therapeutics
House
House Energy & Commerce
House of Representatives
House Ways and Means Committee
Houston
Houston TX
How Full Is Your Bucket?
HPP
HPS
HPS Group
HPS Network
HR 7667
HSAN1E
HSAN1E Society
Human Genome Project
Humanitarian Device Exemption
hunger
Hunter's Hope
Huntersville North Carolina
Huntington's disease
Huntington’s Disease Youth Organization
hurricane dorian
Hurricane Harvey
hurricane ian
hurricane relief
Hyper IgM Foundation
hyper IgM syndrome
hyperphagia
hypoglycemia
hypokalemic periodic paralysis
hypophosphatasia
Hypoplasia
hypotonia
hypoxemia
Hyptonia
I-ORD
IACT
IAMRARE
IAMRARE Patient Registry
IAMRARE Registry
IAMRARE Registry Program
IBM
IBM Watson
ICD Codes
ICER
Ichthyosis
Ilene Sussman
Illinois
Illinois Rare Disease Commission
Immune Deficiency Foundation
immune system
immunoglobulin
immunosuppressed
Impact
Improving Access to Clinical Trials Act
inborn errors of metabolism
Inc.
Inc. v. Becerra
inclusion
Inclusivity
inclusivity policy
Incyte
Incyte Pharmaceuticals
industry
Infantile Myofibromatosis.
Infantile Spasms
infection
infectious disease
Inflation Reduction Act
influencer
infographic
informed conset
infusion coverage
Ingo Helbig
inherited metabolic disorders
Inmazeb
Innovative Research
INSIGHT
INSIGHT: A Global Ocular Melanoma Patient Registry
Insmed
Insmed Inc.
Inspiration
insurance
insurance caps
insurance market protections
insurance-like products
Interferon
internal medicine
International Family Registry for Centronuclear and Myotubular Myopathies
international rare disease day
International Spy Museum
intracranial hypertension
intrahepatic cholangiocarcinoma
Iowa State University
IQVIA
IQVIA Institute
IV infusion therapy
IVQIA
Jack Klugman
Jack Reed
Jacob Haendel’s Recovery Channel
Jacqueline Kraska
Jade Day
Jakafi
Jane Larkindale
Janet Bunge
Janet Woodcock
Janet Woodcock MD
Jann Le Cam
Jansen's
Jansen's Foundation
Jared Polis
Jason Sicklick
Jayne Holtzer Rare Disease Research Grants Program
JDRF
Jefferson University Medical College
Jeffersonian dinner
Jennifer Canvasser
Jennifer Orange
Jennifer's GP House
Jeopardy
Jess Thoene
Jessica Bohonowych
Jill Pollander
Jill Zeigler
Jim Greenwood
Jim Palma
Jimmo v. Sebelius
Jiping Yue
Joe Biden
Joe DiMaggio Children’s Hospital
John Farley
John Hopper
John Jenkins
John K. Jenkins
John Kline
John McCain
John Oliver
John Rista
Johnny Rista
Johns Hopkins Medicine
Johns Hopkins University School of Medicine
Jonathan C. Goldsmith
Jordans Syndrome
José Abdenur
Joseph Farris
Joseph Scheeren
Joseph Shieh
Joshua Frase
Joshua Frase Foundation
joshua mann
journal
JPA
Jr. & Georgia O. Akers Private Foundation
Julia Su
Julia Vitarello
Julie Raskin
Juvenile Rheumatoid Arthritis
Kabuki syndrome
Kam Redlawsk
Kansas Department of Health and Environment
Karen Pignet-Aiach
Kari Luther Rosbeck
karina sturm
KAT6A Foundation
KAT6A Patient Registry
KAT6A syndrome
Kate Bartlett
Kate Obstgarten Family Foundation
Katie Larimore
Katie Thortenson
Kawasaki disease
Kay Holcombe
Keck Graduate Institute
Kedrion P BioPharma Inc.
Kelly Barendt
Kentucky
Kerri Nelson
keynote
Keynote Address
Keynote Speaker
Kira Mann
Kjersti Flatmark
Klaus Romero
Koselugo
krabbe
Krabbe Community United Research and Engagement Study
Krabbe disease
KrabbeConnect
KrabbeCURES
Kristen Angell
Kristin Anthony
Kristin Smedley
KY3
LAM
Langerhans Cell Histiocitosis
Langerhans cell histiocytosis
Lankenau Medical Center
Laura Crandall
Lauren Hill
Lauren Ruotolo
Lauren Wasser
LBSL
LD 972
Leah Lande
leap day
Learning
leber’s hereditary optic neuropathy
Legg Calves Perthes Foundation
legislation
Legislative Document 972
Leigh Syndrome
leiomyosarcoma
Lennox-Gastaut
Lesli Nordstrom
leukemia
Leukemia & Lymphoma Society
Leukemia and Lymphoma Society
leukodystrophy
Levy-Yeboa Syndrome
Levy-Yeboa Syndrome Research
Lewis & Clark College
Lexington Clinic
lgbtq+
LGDA
Li Fraumeni Syndrome
Li Fraumeni Syndrome Association
Liam Cruz
Licensed Practical Nurse
life sciences
Life-Transforming Treatments
Lifetime Achievement Award
Lifetime Television
Light Up for Rare
limited medical assistance
Lindsey Graham
lipidosis
Lipoprotein Lipase Deficiency
Lipoprotein(a) Foundation
Lisa Butler
Lisa Phelps
Lisa Sarfaty
Lisa Terrizzi
Lisa Yue
Listening Sessions
Live Webcast
livestream
Living Rare
Living Rare Forum
living rare living strong
Living Rare Living Stronger
Living Rare Living stronger Forum
Living Rare Living Stronger NORD Patient & Family Forum
Living Rare Living Stronger NORD Patient and Family Forum
living rare living stronger patient & family forum
Living Rare Living Stronger Patient and Family Form
Living Rare Living Stronger Patient and Family Forum
living strong
Living Stronger
Living Stronger NORD Patient and Family Forum
Living Stronger™ NORD® Patient a
Lizzie Valasquez
LMS
Logo
longitudinal
Los Angeles
Los Angeles Times
Lou Gehrig's disease
Louisiana
Loxo Oncology
LPLD
LRLS
LRLSR
Lundbeck
Lundbeck US
LungMAP
Lurie Children's Hospital
Lutheran Services in America
Lymphangioleiomyomatosis
Lymphangiomatosis & Gorham's Disease Alliance
Lymphedema
lymphoma
LYS
Lysogene
lysosomal disease
Lysosomal Storage Diseases
lysosomal storage disorder
lysosomal storage disorders
M.D.
M.S.c.
MA
MacArthur Amendment
Mackenzie's Mission
Maddie's Herd
Madison Shaw
Magenta Therapeutics
Maine
Make a Will
Make a Will Month
Make-A-Wish Foundation
Mallinckrodt Pharmaceuticals
Malonia Aciduria
Malonic Aciduria
MAPkinase pathway
Marathon
Marc Scheineson
March of Dimes
Marcy Toepel
Marfan syndrome
Margaret Hamburg
marginalized
marginalized communities
Maria Kefalas
Marion Finkel
mark anastasio
market reforms
Marketing Approval
Marketplace
Marsha Lanes
Marshall L. Summar
Marshall Summar
Martha Rinker
Mary Dunkle
Mary Lou Retton
Mary Wooten
Mary Wootten
Massachusetts
Massachusetts College of Pharmacy and Health Sciences
Massachusetts General Hospital
Mast Cell Disease
Matthew MIght
Matthew Pearl
matthew zachary
Mauli Ola Foundation
Maurice Klugman
Mayer-Rokitansky-Kuster-Hauser syndrome
mayo-clinic
MCADD
McGill School of Medicine
McGill University Health Centre Research Institute
MD
MD Anderson Cancer Center
MdDS Foundation
MDS
MDS Foundation
ME
Medal
Media
MediaPlanet
Medicaid
Medicaid eligibility
Medicaid enrollment
Medicaid expansion
Medicaid section 1115 waivers
Medicaid/CHIP eligibility
medical
Medical Advisory Committee
Medical Alert Bracelet
medical device
medical devices
Medical Education Advancement
Medical Ethics
Medical Foods
medical foods coverage
medical geneticist
Medical Nutrition
Medical Nutrition Equity
medical nutrition equity act
Medical Product Innovation
medical professionals
Medical research
medical resources
medical reviewers
Medical Students
medically diverse
Medicare
medication refills
medium-chain acyl-CoA dehydrogenase deficiency
Medscape
Megacystis Microcolon Intestinal Hypoperistalsis Syndrome
Melanie Langelier RN
Melvin Van Woert
Member Organizations
Member Spotlight
Members
Members-Only Webinar
Membership Manager
Membership Organizations
Memorial Sloan-Kettering Cancer Center
MEN
MEN-1
mended hearts
Menin
Mental Health
Merced
Mered Parnes
mesothelioma
metachromatic leukodystrophy
Michael A. Pack
Michael Rodgers
Michael Scott
Michael Smedley
Michael's Cause
Michelle Campbell
Mike Porath
Mike Scott
Mila’s Miracle Foundation
military
Milken Institute
Miss America
Miss Connecticut
Miss Iron County
Miss Pre-Teen Connecticut
Miss Utah
MitoAction
MitoAction Mobile
Mitochondrial Disease
Mitochondrial Disorders
Mitochondrial Myopathies
Miyoshi myopathy/limb-girdle muscular dystrophy type 2
MLD
MMA
MMIHS
MMN
Moderna
Mother's Day
Mount Everest
Mount Sinai Hospital
movement disorders
movies
moyamoya
MPN Research Foundation
MPN specialists
MPN-NET/MPN Education Foundation
MPNs
MPS I
MPS I Hurler syndrome
MPSI
MRI
MS
MSKCC
Mt. Washington
MTM
MTM1
Mucopolysaccharidosis IIIA
Mucopolysaccharidosis type 1
multicentric reticulohistiocytosis
multifocal motor neuropathy
Multiple Sclerosis Foundation
Muscular Dystrophy
Muscular Dystrophy Association
myasthenia gravis
mycosis fungoides
myelodysplastic syndromes
Myelofibrosis
myeloproliferative neoplasms
Myhre Syndrome
myositis
myotubular myopathy
Narcolepsy
Narcolepsy Network
narcolepsy type 2
NASA
NASA Astronaut
Nasogastric Tube
Natalia Gomez-Ospina
Natasha Shur
National Brain Tumor Society
National Cancer Institute
National Center for Advancing Translational Sciences
National Center for Advancing Translational Sciences (NCATS)
National Commission on Orphan Diseases
National Defense Authorization Act
National Down Syndrome Congress
National Family Caregivers Month
National Foundation for Ectodermal Dysplasias
National Fragile X Foundation
National Health Council
National Hemophilia Foundation
National Institues of Health
National Institute of Environmental Health Sciences
National Institutes of Health
National Institutes of Health (NIH)
National Institutes of Health (NIH) Undiagnosed Diseases Network (UDN)
National Institutes of Health Undiagnosed Diseases Program
national kidney foundation
National Leiomyosarcoma Foundation
National Marrow Donor Foundation
National MPS Society
National Multiple Sclerosis Society
National Organization for Rare Disorders
national patient advocate foundation
National Pediatric Research Network Act
National Psoriasis Foundation
National Rare Disease or Condition Surveillance System
National Urea Cycle Disorders Foundation
National Volunteer Week
Nationwide Children’s Hospital
Natpara
natural disaster
Natural Histories
natural history
natural history studies
natural history study
Natural History Study Data
Natural History Study Project
Navigating Pediatric ER
Navigating the Pediatric ER
nbs
NBTS
NCATS
NCC Collaborator
Nebraska
NEC
NEC Society
necrotizing enterocolitis
Ned Sharpless
Neena Nizar
NEHI
NEHI Research Foundation
Neil Horikoshi
neonatal intensive care unit
NET cancer
Network
Networking
neurodevelopmental disorders
neuroendocrine cancer
Neuroendocrine Cancers
Neuroendocrine Cell Hyperplasia of Infancy
Neuroendocrine Tumor Research Foundation
Neurofibromatosis Network
Neurofibromatosis Type 1
Neurogene
Neurogenic Orthostatic Hypotension
neurologic
neurology
Neurology Reviews
neuromuscular
Neuromyelitis Optica
Neuromyelitis Optica Spectrum Disorder
New England Journal of Medicine
New Jersey
New York City
New York City Police Department
New York Road Runners
New York University
New York University School of Medicine
New-Onset Refractory Status Epilepticus
newborn
newborn screening
Newborn screening awareness month
Newborn Screening Saves Lives Act
newly diagnosed
Next-Generation Treatments & Advancing Clinical Trials
nf1
NF2
NFL
NGLY1
Nibs
NICU
Niemann-Pick disease type C1
NIH
NIH Clinical Educator of the Year
NIH funding
NIH National Center for Advancing Translational Sciences
NIH research
NIH Resources
NIH Undiagnosed Diseases Program
NKH
NLMSF
NMO
NMO disease
NMOSD
Noah Victoria
nOH
Nominate
nominees
non-medical needs
non-orphan
non-orphan specialty drugs
non-profit leaders
Nonketotic Hyperglycinemia
Nonprofit
nonprofit organizations
nonprofits
Nontuberculous Mycobacterial Lung Disease
Noonan Syndrome
Noonan Syndrome Foundation
NORD
NORD 2022 Rare Disease and Orphan Products Breakthrough Summit
NORD 35th Anniversary
NORD 35th Anniversary Celebration
NORD Board
NORD Board of Directors
NORD COVID-19 Community Survey Report
NORD COVID-19 resource center
NORD Education Team
NORD Event
NORD Founder
NORD Hurricane and Natural Disaster Emergency Relief Fund
NORD IAMRARE Patient Registries
NORD in the News
NORD Medical Advisory Committee
NORD Member Organization
NORD Member Organizations
NORD Members
NORD Online
NORD Patient and Family Forum
NORD Patient Assistance Program
NORD Physician Guides
NORD Policy Team
NORD Position Paper
NORD RAN
NORD Rare Caregiver Respite Program
NORD Rare Disease Video Library
NORD Rare Diseases & Orphan Products Breakthrough Summit
NORD Rare Diseases and Orphan Products Breakthrough Summit
NORD Rare Summit
NORD registry community
NORD Research Grant
NORD Research Grant Program
NORD Resource Guide
NORD Resources
NORD Student Chapter
NORD Student Programs
NORD Students for Rare Club
NORD Summit
NORD survey
NORD Website
NORD's 35th Anniversary
NORD's 35th Anniversary Celebration
NORD's 35th Anniversary Celebration presenting the Rare Impact Awards
NORD's Medical Advisory Committee
NORD's Mission
NORD's Patient Assistance Program
NORD’s Jayne Holtzer Rare Disease Research Grants Program
NORDGivesBack
NORDpod
Norman E "Ned" Sharpless
Norman Winnerman
NORSE
Northeastern University
Northwestern Memorial Hospital
Northwestern University
Northwestern University Feinberg School of Medicine
Northwestern University School of Medicine
Notre Dame
Novartis
Novartis Oncology
Novimmune and Sobi
NTM
NTM Info & Research Inc.
NTM Research & Info
Nulibry
NYC
NYC Marathon
NYS Bill A1470B-2015
NYU
NYU Hospital for Cancer and Blood Disorders
NYU Langone Medical Center
NYU School of Medicine
O. Carter Snead III MD FAAN
Obamacare
obstetrics
Octavio Armenta
ocular melanoma
ODA
ODTC
odyssey
Office of Civil Rights
Office of Hematology and Oncology Products
Office of New Drugs
Office of Orphan Products Development
Office of Rare Diseases Research (ORDR)
OH
Ohio
Oliver S. Eng
Olympian
Omnibus
omnibus spending bill
omphalocele
On Target Laboratories Inc.
oncologist
oncology
Oncology Center of Excellence
OND
online educational tool
online support group
OOPD
op-ed
Open Act
Open Enrollment
Opposition Letter
Oprah Winfrey
Opsoclonus-Myoclonus Syndrome
optic atrophy
or Eosinophilic Esophagitis
Oregon
Organic Acidemia Association
orphan
orphan disease
Orphan Disease Update
Orphan Disease Update and NORD Online newsletter
Orphan Druct Act
orphan drug
Orphan Drug Act
Orphan Drug Act of 1983
Orphan Drug Act Resolution
orphan drug development
Orphan Drug Prices
Orphan Drug Pricing
orphan drug tax credit
orphan drugs
Orphan Drugs in the United States
Orphan Drugs in the United States: Rare Disease Innovation and Cost Trends Through 2019
orphan indications
Orphan products
Orphan Products Grants program
orphan therapies
Orrin Hatch
Oslo University Hospital
Osteogenesis Imperfecta Foundation
Otsuka America Pharmaceutical
Our Curse
Our Odyssey
out-of-pocket
Out-of-pocket costs
Oxbryta
Oxlumo
Pachyonychia Congenitia
PACS1-related syndrome
Pageant
PALS
Pamela Gavin
Pamela K. Gavin
pancreatic cancer
Pancreatic Cancer Action Network
pandemic
Panhematin
PAP
Parade
paraganglioma
parathyroid hormone
Parent
Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophyk Precision Medicine
Paroxysmal Nocturnal Hemoglobinuria
Partha Sen
partial trisomy 8Q duplication syndrome
Partner
Partners for Krabbe Research
Partnership
PassageBio
Passionate
Pat Furlong
Patent
PATH for PWS
patience assistance program
Patience Assistance Programs
Patient
Patient & Family Forum
Patient Access to Investigational Therapies
patient advocacy
Patient Advocate
Patient Advocate Awards
patient advocates
Patient Affairs Staff
Patient AirLift Services
Patient and Caregiver Resource Center
Patient and Family Forum
Patient and State Stability Fund
Patient Assistance
Patient Assistance Program
Patient Assistance Programs
patient care
Patient Community Building
patient data
Patient Engagement
patient engagement listening sessions
Patient Experience Data
Patient Focused
Patient Focused Drug Development Initiative
patient focused drug development meeting
patient journey
patient listening sessions
patient organizations
patient perspective
Patient Perspectives
Patient Powered
Patient Protections
patient registries
patient registry
patient registry data
Patient Service
patient services
patient stories
patient story
Patient Voice
patient-centered
patient-centered outcomes
Patient-Centered Outcomes Research Institute
patient-centric
Patient-First Blueprint
Patient-Focused Drug Development
patient-powered initiatives
Patient/Caregiver Speakers Bureau
patients
Patricia Richardson
Patrick Dunegan
Patrizia Cavazzoni
Patty Murray
Paul Frase
Paul Melmeyer
Paul Melmyer
Paul Newman
Paul Ryan
Paula Mann
pbm
PC Project
PCAST
PCD
PCD Foundation
PCI
PCOR
PCORI
PDSA
PDUFA
PEARLS Project
Pediatric
pediatric cancer
Pediatric ER
pediatric hypophosphatasia
Pediatric Minds Early Childhood Treatment Center
pediatric movement disorders
Pediatric Research Equity Act of 2003
Pediatrics
Pemazyre
Penn State University
Pennsylvania
Pennsylvania Rare Disease Advisory Council
People's Magazine
Perelman School of Medicine
peritoneal mesothelioma
personalized medicine
pet scan
Peter Frampton
Peter L. Saltonstall
Peter Marks
Peter Saltonstall
Petra Kaufmann
PFDD
PFIC
Pfizer
Ph.D.
Pharma
Pharmaceutical Medicine
pharmacies
pharmacy
pharmacy pediatrics club
pharmacy student
PharmaEssentia
PharmaEssentia Corporation
PharmaVOICE
PhD
Phenylketonuria
pheo
pheochromocytoma
PHI
Phil Maderia
Philip John (P.J.) Brooks
Phillip L. Pearl MD
PhRMA
physical therapy
Physician Education
Physician's Guide
Physicians
Pie Fight for PI
pigmented villonodular synovitis
pills
Pitt Hopkins Research Foundation
PKD
PKND
PKU
PKU Organization of Illinois
platelet
Platelet Disorder Support Association
PlatformQ Health
PlatformQ Health Education
PMP
PMP Research Foundation
PNH
poetry
POIS
Policy
Policy 101
Policy and Regulatory Affairs for NORD
Polycythemia Vera
Porphyria
Port Wine Stain Birthmarks
portacath
Portraits of Courage
Portraits of Courage Honoree
Portraits of Courage Honorees
position statement
Positive Exposure Ambassador
Post-Orgasmic Illness Syndrome
Post-Transplant Lymphoproliferative Disease
Post-Transplant Lymphoproliferative Disorder
Poster Abstracts
PPE
Prader-Willi
Prader-Willi Syndrome Association
Pravin Jadhav
Pre-Existing Condition Protections
pre-existing conditions
Pre-Existing Conditions Protections
pregnancy
pregnant
premium
premium assistance
premiums
prescription cost sharing requirements
Prescription Drug Cost Sharing
Prescription drug out-of-pocket cost protections
prescription drug spending
Prescription Drug User Fee Act
prescriptions
President Biden
President Donald Trump
President Obama
President Ronald Reagan
President Trump
Press
press release
pride month
Primary Distal Renal Tubular Acidosis
primary hyperoxaluria type 1
primary immunodeficiency
Primary Myelofibrosis
Primary Orhostatic Tremor
Primary Orthostatic Tremor
Principia Biopharma
Principles for Healthcare Coverage Reform
PRISMS
Proclamation
product review
Professor David Morris
Progeria
Project Baby Bear
project n95
Project RDAC
Promising Pathway Act
Protect Patients Now
protect your healthcare coverage
proteus syndrome
Providence Marathon
PRV Program
PSA
Pseudomyxoma Peritonei
Psychology Today
PTEN
PTEN hamartoma tumor syndrome
PTEN Hamartoma Tumor Syndrome Foundation
PTLD
public health
public health challenge
Public Health Leadership Award
Public Policy
Public Service Announcement
public-private partnership
pulmonary
pulmonary hypertension
Pulmonary Hypertension Association
Pulmonary Nontuberculous Mycobacterial Disease
pulmonary valve stenosis
pulmonology
pyknodysostosis
Pyruvate Kinase Deficiency
Q&A
Quincy M.E.
QuintilesIMS Institute
quotes
R&D
Rachel Sher
Rachid Tazi-Ahnini
Radium Hospital
Raise Awareness
Raise Your Hand Campaign
Raise Your Hand to Fight Rare Disease Research Campaign
Rally for Medical Research Hill Day
Ramaprasad Srinivasan MD
RAN
RAN State Ambassador
randi clites
Randy Rogers Band
Rapid Response Leadership Series
Rapid Response Seed Grant
Rare
Rare Access
RARE Act
Rare Action
Rare Action Netowrk
Rare Action Network
Rare Action Network State Ambassador
Rare Action Road Tour
Rare and Neglected Diseases Caucus
Rare and Undiagnosed Network (RUN)
Rare As One
Rare As One Project
rare autoimmune diseases
rare birth defect
rare blood disease
rare cancer
Rare Cancer awareness
rare cancer coalition
rare cancer day
Rare Cancer Day 2021
Rare Cancer Day 2022
Rare Cancer Foundation
rare cancer patient stories
rare cancers
Rare Caregiver Respite Program
rare disease
Rare Disease Advancemenet Research and Education Act
Rare Disease Advisory Council
Rare disease advisory councils
Rare Disease Advocacy
Rare Disease Advocates
Rare Disease Awareness
Rare Disease Clinical Research Network
Rare Disease Club
rare disease community
Rare Disease Congressional Caucus
Rare Disease Continuing Medical Education
rare disease cures
Rare Disease Cures Accelerator
Rare Disease Cures Accelerator Data and Analytics Platform
Rare Disease Database
Rare Disease Day
Rare Disease Day 2015
Rare Disease Day 2016
Rare Disease Day 2018
Rare Disease Day 2019
Rare Disease Day 2019. Rare Action Network
rare disease day 2022
Rare Disease Day 2023
Rare Disease Day U.S. Sponsor
Rare Disease Day US
Rare Disease Diversity Coalition
rare disease drug development
Rare Disease Education
Rare Disease Educational Support Program
rare disease facts
Rare Disease Funding Opportunities
Rare Disease Institute
Rare Disease Institute at Children's National Health System
rare disease nonprofits
Rare Disease of the Day
Rare Disease of the Week
rare disease patient
Rare Disease Patient & Caregiver Resource Center
rare disease patients
Rare Disease Pediatric Review Voucher Program
Rare Disease Report
Rare Disease Reports
Rare disease research
Rare Disease Research Grant
Rare Disease Research Grant Program
Rare Disease Respite
Rare Disease Summit
rare disease technology
rare disease therapies
rare disease treatments
Rare Disease Video Library
Rare Disease Videos
Rare Diseases
Rare Diseases & Orphan Products Breakthrough Summit
Rare Diseases Act of 2001
Rare Diseases Act of 2002
Rare Diseases and Orphan Products
Rare Diseases and Orphan Products Breakthrough Summit
Rare Diseases and Orphan Products Brekathrough Summit
Rare Diseases Clinical Research Network
Rare Diseases Clinical Research Network (RDCRN)
Rare Diseases Europe
Rare Diseases International
Rare Diseases Report: Cancers
Rare Diseases Report: Rheumatology
Rare Disesase Day
rare disorder
Rare Disorder Survey
Rare Disorder Toolkit
rare disorders
rare genetic cancer syndrome
rare genetic disease
Rare Impact
Rare Impact 2017
Rare Impact Award
Rare Impact Award Honoree
Rare Impact Award Honorees
Rare Impact Award Nominations
Rare Impact Awards
Rare Impact Awards Ceremony
rare lysosomal storage disorder
Rare Mom
Rare Moms
rare neurological condition
Rare Neurological Disease Special Report
rare neuromuscular and neurodegenerative diseases
Rare Pediatric Disease Priority Review Voucher
Rare Pediatric Disease Priority Review Voucher Program
Rare Revolution Magazine
Rare Spotlight
Rare Summit
RareAction
RareAction Network
RareCancer
RareCare
RareCare Patient Assistance
rareconnect
rareedu
RareInsights
RareLaunch
Ray Stevens
RCD 22
RCD'21
RDAC
RDACs
rdca
RDCA-DAP
RDCRN
RDD
RDD19 Story
RDDC
RDDNIH
RDEB
RDI
Reactive Arthritis
rebates
Rebecca Aune
Rebecca Campbell
Rebekah Bressi
Recap
Recessive Dystrophic Epidermolysis Bullosa
Recommended Uniform Screening Panel
Reconciliation
Reconstructive Surgery Act of 2005
Recurrent Respiratory Papillomatosis
Red Cross
Reed's Syndrome
Reflex Sympathetic Dystrophy
Regeneron
Regis College
Registered Nurse
registries
Registry
registry model
registry program
Regsitry
regulatory affairs
regulatory agencies
relief program
Remember the Girls
Renal Cell Cancer
Rep. DeGette
Rep. Upton
Report Card
Representative Bilirakis
Representative Butterfield
Representative Degette
Representative Henry Waxman
Representative Lance
Representative Margaret Craven
Representative Upton
Request for Proposal
Request for Proposals
Requests for Proposals
research
Research Director
Research Fund
Research Funding
research grant
Research Grants
Research Ready
research study
Research! America
ResearchMatch
resource
resources
Respite Care
Retaining Access and Restoring Exclusivity Act
Retevmo
Rethymic
Retinal Vasculopathy with Cerebral Leukodystrophy
Retinitis Pigmentosa
Retrophin
Rett Syndrome
Revised Senate Bill
RFP
RFPs
rheumatology
RIA
Rice University
Ricki Lewis
Ridgeback Biotherapeutics
Right to Try
Right to Try Act
Robert Califf
Robert Chen
Robert Long
Robert Lutz
Robert Rosenbloom
Rochester Institute of Technology
Rochester Minnesota
Rock and Roll Hall of Fame
RocketPharma
Ronald Reagan
Roscoe Brady
Rowan
Rozlytrek
Ruling
RUN
Running
Running for Rare
Running for Rare Diseases
Running for Rare Program
Running on Air
Running4Rare
Rush University Hospital
Rush University Medical Center
RUSP
Russ Illes
RVCL
Rx
Ryplazim
RYR-1 Foundation
RYR-1 gene
RYR-1 Related Diseases
S 4348
S. 201
S. 204
S.1878
safe step act
Samantha Scheer
Sanfilippo Syndrome
Sanfillipo Syndrome
Sanika Rane
Sanofi Genzyme
Sara's Cure
Sarah A. Taylor
Sarcoidosis
sarcoma
Savannah Hollis
Save Orphan Drugs
Saving Eliza
SB 7
scd
SCDAA
schools
Schuurs-Hoeijmakers Syndrome
scleroderma
Scleroderma Research Foundation
SCN2A
SCN2A Disorder
SCN8A Survey
SCOTUS
Sean Hepburn Ferrer
Seattle Children's Hospital
Secretary Azar
Secretary of the Department of Health and Human Services
Section 1115 waivers
SELENON/SEPN1-related myopathy
Senate
senate briefing
Senate Cures
Senate Cures Legislation
Senate Finance Committee
Senate Health Education Labor and Pensions Committee
Senate Healthcare Bill
Senate Innovations for Healthier Americans Initiative
Senator
Senator Bill Cassidy
Senator Brown
Senator Edward Kennedy
Senator Elizabeth Warren
Senator from Washington
Senator Hatch
Senator Michael Bennet
Senator Nancy Kassebaum
Senator Orrin
Senator Orrin Hatch
Senator Richard Blumenthal
Senator Richard Burr
Senator Roberts
Senator Tammy Baldwin
Senator Ted Kennedy
September 11th
September 30
Sequestration
Seth Rotberg
Severe Combined Immune Deficiency (SCID-ADA)
severe weather
SGS
Shafali Spurling Jeste
Shaquille O'Neal
Share Your Story
Sheba Medical Center
Shel Silverstein
Sheldon Schuster
sherrod brown
Shine Light on Rare Diseases
Shire
Short Bowel Syndrome
Shortage
Show Your Rare
Show Your Stripes
Show Your Stripes Challenge
Shprintzen Goldberg Syndrome
sickle cell disease
Sickle Cell Disease Association of America
SIDS
Sign up today
Sika Dunyoh
Silicon Valley Community Foundation
Silver Sponsor
Simran Handa
Sing me a song
Sitosterolemia
Sitosterolemia Foundation
skill
Skin Disorders
SLC6A1
SLC6A1 Connect
Sleep
Sly syndrome
SMA
SMS
snowstorms
Snyder-Robinson Syndrome
social distancing
social media
Social Security Administration
social support
software platforms
Sophia Hanson
South Carolina
space center houston
spacticity
Special Books by Special Kids
special education
special enrollment periods
Special Moments
Special Olympics
specialty drug spending
Spectrum vs. Burwell
spencer health solutions
Spending Bill
Spinal Muscular Atrophy
Spinocerebellar Ataxia
Spinocerebellar ataxia autosomal recessive 9 (SCAR9)
Sponsor
Sprengell Deformity
SPS
SRS
ssa
SSI
St. Baldrick’s Foundation
standards of care
Stanford University
STAR Act
STAT
state
State Advocacy
State Ambassador
state capitol
state government
state governments
state health departments
state house
State House Event
State House Events
state issues
State Legislation Tracker
State Policy
State Policy Legislative Tracker
State Progress Report
State Report Card
State Report Carfd
State Representative from Massachusetts
state-by-state
state-regulated health insurance
Stealth BioTherapeutics
stem cell transplant
step therapy
Stephanie Cindric
Stephen Cederbaum
Stephen Friedhoff
Stephen Groft
Stephen Hahn
Steven Grossman
stiff body syndrome
Stiff Person Syndrome Research Foundation
stiff persons syndrome
stitches
Stripe Out Your School
stroke
Strong
Strongbridge
Stronger Scholarship
student leaders
Student Membership
Students
Students for Rare
Students for Rare Chapter
study
stupid cancer
stupid cancer show
Sturge Weber Foundation
Sturge-Weber Syndrome
STXBP1
STXBP1 encephalopathy
STXBP1 Foundation
STXBP1 Move to Cure
sub-study
SUDC
Sudden Infant Death Syndrome
Sudden Unexpected Death Data Enhancement and Awareness Act (H.R. 669)
sudden unexplained death in childhood
Suddenly Sleepy Saturday
Sudhir Borgonha
Summer Camp
Summit
SUOX mutation
Supplemental Security Income
Support
Supreme Court
surprise medical bills
Survey
survey findings
Susan A. Berry
Susan Fernback
Susan G. Komen
Sylvia Burwell
symptoms
Syringomyelia
systemic scleroderma
SZB
Tai Spargo
take action
Takeda
TANGO2 disorder
TANGO2 Research Foundation
Tara Notrica
target cancer foundation
TargetCancer Foundation
Tatton Brown Rahman Syndrome
Tatton Brown Rahman Syndrome Community
tax credit
Tax Cut and Jobs Act
Tax Cuts and Jobs Act
tax reform
Taylor Kane
TBRS
TBRS Community
TCS
TCS New York City Marathon
Teachers
TEDx
telehealth
Telehealth Extension and Evaluation Act
telehealth principles
telehealth services
telemedicine
Telomore
Tepezza
Terry Jo Bichell PhD
Texas
Texas Children's Hospital
Texas Rare Action Network
Texas Rare Action Network State Ambassador
Texas State Ambassador
Thank You
Thanksgiving
The 100 Days Agenda: A Patient-First Blueprint
The Adrenoleukodystrophy (ALD) Foundation
The ALS Association
The Association for Creatine Deficiencies
The Atypical HUS Foundation
The Avalon Foundation
The Awesome Disease
The Balancing Act
The Calliope Joy Foundation
The Children’s Hospital of Philadelphia
The Chondrosarcoma Foundation
The Critical Path Institute
The Cute Syndrome Foundation
The David Ashwell Foundation
The David Bach Consort
The Dup15qAlliance
The Galactosemia Foundation
The Guthy-Jackson Charitable Foundation
The Hill
The Hole in the Wall Gang Camp
The Honorable Michael McCaul
The Honorable Michael McCual
The Honorable Sarah Davis
The Hope Fund
The Legacy of Angels Foundation
The Marfan Foundation
The Mighty
The Moebius Syndrome Foundation
The Morgan Leary Vaughan Fund
The Motley Fool
The NORD Guide to Rare Disorders
The OMSLife Foundation
The Physician’s Guide
The Power of Patients
The Power of Patients: Informing Our Understanding of Rare Diseases
The Rare Disease Cures Accelerator-Data and Analytics Platform
The Recessionals
The Snow Foundation
The Snyder-Robinson Syndrome Foundation
The United Mitochondrial Disease Foundation
The University of Chicago
The University of Sheffield
The VEDS Movement
The Week
TheMighty.com
therapeutic recreation
Therapeutics for Rare and Neglected Diseases (TRND)
therapy
Theresa Mullin
Theresa Mullin PhD
ThinkGenetic
Thoma Family
Thomas Jefferson
Thomas M. Haberman
tik tok
Tim Boyd
Timothy Yu
titanium rib
TK2 Mitochondrial DNA Depletion Syndrome
Together We Are Strong
Tom Rath
tom rhoads
Tom Rusin
Tony Ferrandino
toolkit
Tourette Syndrome Foundation
toxic acute progressive leukoencephalopathy
Toxic Shock Syndrome
tracheobronchomalacia
Travere Therapeutics
Travis Flores
treatment
treatment protocols
Trevor Noah
triaging
TRICARE
Trikafta
Trio Health
Tristan Lee
Trump Administration
TSC New York City Marathon
TSS
TUBB4A gene mutation
tuberous sclerosis complex
tumor
tumors
Turalio
Turner Syndrome
Tweetchat
twins
Twitter
U.S. Court of Appeals
U.S. Food and Drug Administration
U.S. House of Representatives
U.S. Olympian
U.S. Senate
UCLA
UDN
UFA
Ulcerative Colitis
Ultragenyx Pharmaceutical Inc
UMass Memorial Hospital
UMDF
underrepresented populations
Undiagnosed
Undiagnosed Day
Undiagnosed Diseases
Undiagnosed Diseases Network
Undiagnosed Diseases Patient Assistance Program
Undiagnosed Diseases Program (UPD)
undiagnosed patients
Undiagnosed Patients Registry
undiagnosed registry
United Healthcare
United Leukodystrophy Foundation
United Mitochondrial Disease Foundation
United States
United States Senate
United Therapeutics Corporation
University Health Network
University Hospital of Pisa
University of California
University of California Los Angeles
University of California San Francisco
University of Manitoba
University of Maryland
University of Maryland School of Pharmacy
University of Michigan
University of Milan
University of Minnesota
University of Notre Dame
University of Pennsylvania
University of Wyoming
University of Zurich
unmet need
Urea Cycle Disorders
US
US Congress
US Department of Health and Human Services
US Food and Drug Administration
US Food and Drug Administration Commissioner
US Hereditary Angiodema Association
US News and World Report
user fee
user fee package
user fee reauthorization
user fees
Utah
uveal melanoma
vaccination prioritization
vaccine
vaccines
Vanda
Vanderbilt Kennedy Center for Excellence in Developmental Disabilities
Vanderbilt University
Vanessa Boulanger
Vanessa Devore
Vascular Ehlers-Danlos Syndrome
Vasculitis
VCP
Venture Philanthropy
VEPTR
Vermont
Vertex Pharmaceuticals
Vertical Expandable Prosthetic Titanium Rib
veto
VHL
VHL Alliance
Vice President
Vice President of Regulatory and Government Affairs
video
video submissions
videos
Violins for Vasculitis
viral videos
Virginia
virtual event
Virtual Hill Day
virtual platforms
Virtual Race
virtual tools
Voice of America
voice of rare disease
Voice of the Patient
Voice of the Patient Report
Voices of Rare Cancer
volunteer
von Hippel-Lindau
Waldenstrom Macroglobulnemia
Walker-Warburg syndrome
Warner Theatre
Washington D.C.
Washington DC
Washington Post
Washington State University
Washington University School of Medicine
Ways and Means
Webinar
WebMD
WEGO Health
Wellstat Therapeutics Corporation
Wendy Chung MD PhD
Westminster College
White House
white paper
whole exome sequencing
whole genome sequencing
Wilbur Bowne
Wilhelm Foundation
will
William & George Akers Private Foundation
William Akers
William Cruz
William Yank
Williams Syndrome
Wipro
Wiskott-Aldrich Syndrome
WJLA-TV
Wolfram Syndrome
workshop
world blood donor day
World Cholangiocarcinoma Day
World Health Day
World Orphan Drug Congress
worldwide collaboration
Worldwide Syringomyelia & Chiari Task Force
Worldwise Syringomyelia & Chiari Task Force
Worth magazine
WSCTF
WWS
www.rarediseases.org
X International Pemphigus & Pemphigoid Foundation
X-linked diseases
X-linked myotubular myopathy
Xavier Becerra
xeroderma pigmentosum
XLH Network Inc.
Yair Ankster
Yann Le Cam
young adult rare disease
Young Investigator Grant
Zafgen
Zavesca
zebra
Zika
Zokinvy
Zolgensma
zollinger-ellison syndrome
Living Rare, Living Stronger Forum
Posted April 23, 2020 by NORD
Categorized in -
Uncategorized
NORD’s Director of Research Programs Publishes Article on Rare Disease Research in the 2020 Rare Neurological Disease Special Report
The 2020 Living Rare, Living Stronger Forum Will Now Be Presented As A Virtual Event
National Organization for Rare Disorders
Your Name
(Required)
Your Email
(Required)
I show my stripes for...
(Required)
Max. 5 words
Photo
(Required)
Max file size 5MB. Min. image size 260px X 260px
Drop files here or
Select files
Max. file size: 5 MB.
Your Message
(Required)
Max. 15 words
Consent
(Required)
I agree to the terms and conditions
In these Standard Terms and Conditions, “Your Content” refers to any audio, video, text, images, or other material you provide or display. By providing “Your Content”, you grant NORD a license to read, use, reproduce, adapt, modify, publish, translate, and distribute the content in our marketing materials and to all media. “Your Content” must be your own and must not invade any third‐party’s rights. When you submit content or information to NORD, you are allowing the public to access and use that information, and to associate it with you. NORD reserves the right to remove “Your Content” at any time, without notice. We take your privacy very seriously. Subject to the Terms and Conditions above, NORD will never sell or disclose your personal information.
Name
This field is for validation purposes and should be left unchanged.
×
Name
(Required)
First
Last
Email
(Required)
Enter Email
Confirm Email
Photos
Drop files here or
Select files
Accepted file types: jpeg, png, jpg, Max. file size: 3 MB.
Comments
(Required)
Please let us know what's on your mind. Have a question for us? Ask away.
Phone
This field is for validation purposes and should be left unchanged.
×
Name
(Required)
First
Last
Email
(Required)
Enter Email
Confirm Email
Comments
(Required)
Please let us know what's on your mind. Have a question for us? Ask away.
Comments
This field is for validation purposes and should be left unchanged.
×
