About Us
Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.
Muscular Dystrophy Association Statement to our Community
New York – Friday, July 18, 2025 – We are heartbroken by the recent loss of an MDA community member in a gene therapy trial for limb-girdle muscular dystrophy (LGMD). Our deepest condolences go out to loved ones and to all in the LGMD community who are grieving.
We also recognize concerns in the Duchenne muscular dystrophy (DMD) community following news that the FDA has asked to pause shipments of Elevidys after two adolescent patients died earlier this year. These developments are painful, and they’ve shaken the hope that many families have carried for years.
Gene therapy remains one of the most hopeful frontiers in neuromuscular research. These painful losses remind us how complex and fragile progress can be and how essential it is that safety remains at the center of every step forward. We’re encouraged to see the FDA working closely with researchers, companies, and patient advocates to strengthen safeguards and ensure responsible momentum across all emerging treatments.
MDA will continue to work with the pharmaceutical industry and FDA for safe, effective options. MDA’s mission remains as clear as ever: we believe in the future of gene therapy, but our commitment to safety is non-negotiable. We are here to support every person and family affected by LGMD and DMD as we continue pushing forward together.
Families may contact the MDA Resource Center for guidance and support at 1-833-ASK-MDA1 or email ResourceCenter@mdausa.org.
Media contact press@mdausa.org.
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, Bluesky, TikTok, LinkedIn, and YouTube
About Muscular Dystrophy Association’s 75th Anniversary
In 2025, the Muscular Dystrophy Association proudly marks 75 years legacy, impact and momentum in the fight against neuromuscular diseases. Since our founding, MDA has been at the forefront of research breakthroughs, providing access to comprehensive care, and championing the rights of people living with muscular dystrophy, ALS, and over 300 other neuromuscular diseases. This milestone has been made possible by generations of dedicated support from people living with neuromuscular disease, their families, researchers, clinicians, volunteers, and donors—who boldly drive our mission forward. As we look ahead, we remain committed to honoring this legacy, building on the impact we’ve made together, and continuing our momentum toward transformative progress for people living with neuromuscular disorders. Learn more at MDA75.org.
