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Clinical Research

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. MDA’s mission is to empower the people we serve to live longer, more independent lives. As part of MDA’s mission, we are dedicated to ensuring the neuromuscular disease community is aware of ongoing and available research, including clinical trials and observational studies.

Below you can access a list of trials and studies that MDA is aware are currently enrolling participants. Please note, MDA does not conduct these trials or studies; however, we gather the information and present it in order for the information to be more readily accessible to the community. Click on the link to learn about a trial or study of interest and to help you understand if you are eligible.

Additional resources:

Visit MDA's Clinical Trial finder
Contact MDA Resource Center at 1-833-ASK-MDA1 (1-833-275-6321) or ResourceCenter@mdausa.org

Disease Area Quest Blog Link Phase Sponsor
Amyotrophic lateral sclerosis (ALS) AMX0114 Phase 1 Amylyx Pharmaceuticals, Inc.
Becker muscular dystrophy (BMD) Natural History Study Natural History Study University of Florida
Becker muscular dystrophy (BMD), Duchenne muscular dystrophy (DMD) Research Study Research Study CureDuchenneLink ®
Duchenne muscular dystrophy (DMD) SRD-001 (AAV1/SERCA2a) Phase 1b / 2a Sardocor Corp
Duchenne muscular dystrophy (DMD) Observational Study Observational University of Delaware
Becker muscular dystrophy (BMD), Duchenne muscular dystrophy (DMD) Research Study Research Study CureDuchenneLink ®
Duchenne muscular dystrophy (DMD) RGX-202 Phase 3 REGENXBIO
Duchenne muscular dystrophy (DMD) NS-089/NCNP-02-201 Phase 2 NS Pharma
Duchenne muscular dystrophy (DMD) Observational Study Albany Medical College
Duchenne muscular dystrophy (DMD) SGT-003 Phase 1/2 Solid Biosciences
Friedreich's Ataxia (FA) Observational Lexeo Therapeutics
Limb-girdle muscular dystrophy (LGMD) AB-1003 Phase 1/2 AskBio
Myasthenia gravis (MG) Genome-wide association studies (GWAS) Research Study George Washington University
Myasthenia gravis (MG) Cladribine Phase 3 EMD Serono
Myotonic Dystrophy DYNE-101 Phase 1/2 Dyne Therapeutics
Myotonic Dystrophy SAR446268 Phase 1 Sanofi
Myotonic Dystrophy Natural History Study Natural History Study Sanguine Biosciences
Myotonic Dystrophy Natural History Study University of Rochester
Spinal muscular atrophy (SMA) Risdiplam (Evrysdi) Observational Genentech
Spinal muscular atrophy (SMA) Risdiplam Phase 4 Genentech, Inc.
Spinal muscular atrophy (SMA) Research Study Natural History Study Ohio State University
Spinal muscular atrophy (SMA) Observational Study Observational Natera

MDA Resource Center: We’re Here For You

Our trained specialists are here to provide one-on-one support for every part of your journey. Send a message below or call us at 1-833-ASK-MDA1 (1-833-275-6321). If you live outside the U.S., we may be able to connect you to muscular dystrophy groups in your area, but MDA programs are only available in the U.S.

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