We deeply appreciate our clinical trial participants and their role in advancing treatments for pediatric neuromuscular disorders. We aim to provide a child-friendly, personalized experience and are committed to supporting families with timely information and responsive care throughout the trial process..
- Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: SGT-003 Sponsors: Solid Biosciences Inc. Recruiting
- Conditions: Duchenne Muscular Dystrophy Interventions: Drug: Givinostat Sponsors: Italfarmaco; Cromsource Recruiting
- Conditions: Muscular Dystrophy, Duchenne Interventions: Genetic: delandistrogene moxeparvovec Sponsors: Sarepta Therapeutics, Inc.; Hoffmann-La Roche Recruiting
- Conditions: Duchenne Muscular Dystrophy Interventions: Drug: SRP-4045; Drug: SRP-4053; Drug: Placebo Sponsors: Sarepta Therapeutics, Inc. Completed
- Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: delandistrogene moxeparvovec; Procedure: Plasmapheresis Sponsors: Sarepta Therapeutics, Inc. Terminated
- Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: delandistrogene moxeparvovec; Genetic: placebo Sponsors: Sarepta Therapeutics, Inc.; Hoffmann-La Roche Active, not recruiting
- Conditions: Spinal Muscular Atrophy; Neuromuscular Diseases; SMA Interventions: Drug: taldefgrobep alfa; Drug: Placebo; Drug: taldefgrobep alfa Sponsors: Biohaven Pharmaceuticals, Inc. Active, not recruiting
- Conditions: Spinal Muscular Atrophy Interventions: Biological: onasemnogene abeparvovec-xioi Sponsors: Novartis Gene Therapies; PRA Health Sciences Completed
- Conditions: Spinal Muscular Atrophy; Spinal Muscular Atrophy Type 3; Spinal Muscular Atrophy Type 2; SMA; Neuromuscular Diseases; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal; Neuromuscular Manifestations; Anti-myostatin Interventions: Drug: Apitegromab; Drug: Placebo Sponsors: Scholar Rock, Inc. Completed
- Conditions: Pompe Disease Interventions: Drug: S-606001; Drug: Placebo Sponsors: Shionogi Recruiting
- Conditions: Pompe Disease (Late-onset) Interventions: Biological: Cipaglucosidase Alfa; Drug: Miglustat Sponsors: Amicus Therapeutics Active, not recruiting
If your child doesn’t qualify for a trial at our site, we may be able to help you find one elsewhere. For details on current studies, please email us.
Additional resources
NIH ClinicalTrials.gov
Complete listing of studies tracked by the National Institutes of Health