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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) MyoRegulator® Feasibility Study PathMaker Neurosystems Inc
Amyotrophic lateral sclerosis (ALS) ALS Smartphone Application Observational Johns Hopkins University School of Medicine
Amyotrophic lateral sclerosis (ALS) ION363 Phase 3 IONIS Pharmaceuticals
Amyotrophic lateral sclerosis (ALS) Tofersen Phase 3 Biogen
Becker muscular dystrophy (BMD), Duchenne muscular dystrophy (DMD) Research Study Research Study CureDuchenneLink ®
Becker muscular dystrophy (BMD) Vamorolone Phase 2 University of Pittsburgh
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
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
Duchenne muscular dystrophy (DMD) Research Study Research Study Virginia Commonwealth University
Duchenne muscular dystrophy (DMD) Survey Research Study Rice University
Duchenne muscular dystrophy (DMD) Oral Ifetroban Phase 2 Cumberland Pharmaceuticals Inc.
Friedreich's Ataxia (FA) LX2006 Phase 1/2 Lexeo Therapeutics, Inc.
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) Gefurulimab Phase 3 Alexion AZ Rare Disease
Myasthenia gravis (MG) Cladribine Phase 3 EMD Serono
Myasthenia gravis (MG) Descartes-08 Phase 1/2 Cartesian Therapeutics
Myasthenia gravis (MG) Pozelimab and Cemdisiran Phase 3 Regeneron
Myotonic Dystrophy Natural History Study Natural History Study Sanguine Biosciences
Myotonic Dystrophy Natural History Study University of Rochester
Myotonic Dystrophy Survey Newcastle University, U.K.
Pompe disease ATB200/AT2221 Phase 3 Amicus Therapeutics
Spinal muscular atrophy (SMA) Observational Study Observational Natera
Spinal muscular atrophy (SMA) Risdiplam (Evrysdi) Observational Genentech
Spinal muscular atrophy (SMA) Survey Research Study Saint Elizabeth University
Spinal muscular atrophy (SMA) Research Study Natural History Study Ohio State 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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