Clinical Trials
Disease Area | Study 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) | 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 |
Charcot-Marie-Tooth disease (CMT) | Study | Natural History Study | Ohio State University |
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) | Ataluren | Phase 2 | Rare Disease Research |
Duchenne muscular dystrophy (DMD) | Eteplirsen | Phase 3 | Rare Disease Research, LLC |
Duchenne muscular dystrophy (DMD) | WVE-N531 | Phase 1b / 2a | Wave Life Sciences |
Duchenne muscular dystrophy (DMD) | Oral Ifetroban | Phase 2 | Cumberland Pharmaceuticals |
Myasthenia gravis (MG) | Pozelimab and Cemdisiran | Phase 3 | Regeneron |
Myasthenia gravis (MG) | Descartes-08 | Phase 1/2 | Cartesian Therapeutics |
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) | 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.