
These groups—many founded by parents within the rare disease community—help connect patients and families to education, research, and support services. Here are just a few of the organizations that work to raise both awareness of rare diseases and funds directed toward research while supporting their communities.
Muscular Dystrophy Association
Parent Project Muscular Dystrophy
Cure Duchenne
Jett Foundation
Little Hercules Foundation
Team Joseph
Charley’s Fund
Hope for Gabe
Joining Jack
Duchenne UK
Cure Spinal Muscular Atrophy
Friedreich’s Ataxia Research Alliance
MTM CNM Family Connection
Joshua Frase Foundation
Barth Syndrome Foundation
Canavan Foundation
Chasing Connor’s Cure
Asociación Española de Enfermos de Pompe
Cure Congenital Muscular Dystrophy
Charcot-Marie-Tooth Association
National Ataxia Foundation
United Mitochondrial Disease Foundation (UMDF)
Myasthenia Gravis Foundation of America
RYR-1 Foundation