BREAKING NEWS: New drug approved by the FDA for Duchenne muscular dystrophy. Learn more about this new treatment option.

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) Radicava Observational Mitsubishi Tanabe Pharmaceutical America (MTPA)
Amyotrophic lateral sclerosis (ALS) ALS Smartphone Application Observational Johns Hopkins University School of Medicine
Amyotrophic lateral sclerosis (ALS) Pegcetacoplan Phase 2 Apellis Pharmaceuticals
Amyotrophic lateral sclerosis (ALS) BIIB105 Phase 1/2 Biogen
Amyotrophic lateral sclerosis (ALS) ION363 Phase 3 IONIS Pharmaceuticals
Amyotrophic lateral sclerosis (ALS) Tofersen Phase 3 Biogen
Amyotrophic lateral sclerosis (ALS) MyoRegulator® Feasibility Study PathMaker Neurosystems Inc
Amyotrophic lateral sclerosis (ALS) Remote Monitoring Digital Study Observational Mitsubishi Tanabe Pharma America, Inc.
Amyotrophic lateral sclerosis (ALS) Study Phase 3 Cytokinetics, Inc.
Amyotrophic lateral sclerosis (ALS) Study Research Study Mitsubishi Tanabe Pharma Holdings America, Inc. (MTHA)
Becker muscular dystrophy (BMD) Vamorolone Phase 2 University of Pittsburgh
Becker muscular dystrophy (BMD) EDG-5506 Phase 2 Edgewise Therapeutics
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 ®
Becker muscular dystrophy (BMD) Vamorolone Phase 2 University of Pittsburgh
Charcot-Marie-Tooth disease (CMT) Study Natural History Study Ohio State University
Dermatomyositis Brepocitinib Phase 3 Priovant Therapeutics
Dermatomyositis Ravulizumab Phase 2 Alexion Pharmaceuticals
Dermatomyositis IgPro20 Phase 3 CSL Behring
Duchenne muscular dystrophy (DMD) Research Study Observational University of Florida
Duchenne muscular dystrophy (DMD) Research Study Research Study Virginia Commonwealth University
Duchenne muscular dystrophy (DMD) Survey Phase 3 University of Rochester
Duchenne muscular dystrophy (DMD) Survey Research Study Rice University
Duchenne muscular dystrophy (DMD) Oral Ifetroban Phase 2 Cumberland Pharmaceuticals Inc.
Duchenne muscular dystrophy (DMD) Ifetroban Phase 2 Cumberland Pharmaceuticals Inc.
Duchenne muscular dystrophy (DMD) Observational Study Observational University of Delaware
Duchenne muscular dystrophy (DMD) Ataluren Phase 2 Rare Disease Research
Duchenne muscular dystrophy (DMD) Eteplirsen Phase 3 Rare Disease Research, LLC
Becker muscular dystrophy (BMD), Duchenne muscular dystrophy (DMD) Research Study Research Study CureDuchenneLink ®
Duchenne muscular dystrophy (DMD) Brogidirsen Phase 2 NS Pharma
Duchenne muscular dystrophy (DMD) Observational Study Observational Center for Health + Technology at the University of Rochester
Duchenne muscular dystrophy (DMD) WVE-N531 Phase 1b / 2a Wave Life Sciences
Duchenne muscular dystrophy (DMD) Oral Ifetroban Phase 2 Cumberland Pharmaceuticals
Friedreich's Ataxia (FA) LX2006 Phase 1/2 Lexeo Therapeutics, Inc.
Myasthenia gravis (MG) Pozelimab and Cemdisiran Phase 3 Regeneron
Myasthenia gravis (MG) Descartes-08 Phase 1/2 Cartesian Therapeutics
Myasthenia gravis (MG) Batoclimab (IMVT1401) Phase 3 Immunovant
Myasthenia gravis (MG) Genome-wide association studies (GWAS) Research Study George Washington University
Myasthenia gravis (MG) Study Observational Worrell
Myasthenia gravis (MG) ALXN2050 Phase 2 Alexion Pharmaceuticals
Myotonic Dystrophy Pitolisant Phase 2 Harmony Biosciences
Myotonic Dystrophy Survey Newcastle University, U.K.
Neuromuscular disease (NMD) ASP0367 (MA-0211) Phase 2 Astellas
Neuromuscular disease (NMD) Study Natural History Study Icahn School of Medicine at Mount Sinai
Neuromuscular disease (NMD) Tideglusib / AMO-02 Phase 2 AMO Pharma Ltd
Neuromuscular disease (NMD) Study Natural History Study University of Florida
Neuromuscular disease (NMD) Study Observational University of California, Davis Neuromuscular Research Lab
Pompe disease ATB200/AT2221 Phase 3 Amicus Therapeutics
Spinal muscular atrophy (SMA) Research Study Natural History Study Ohio State University
Spinal muscular atrophy (SMA) Research Study Research Study Division of Pediatric Neurology at the University of Iowa
Spinal muscular atrophy (SMA) Research Study Research Study Brandeis University
Spinal muscular atrophy (SMA) Research Study Research Study Sanguine Biosciences
Spinal muscular atrophy (SMA) Study Natural History Study Russel Sage College
Spinal muscular atrophy (SMA) Survey Research Study Saint Elizabeth University

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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