Additional Neuromuscular Disorders

Jerry R. Mendell, MD, Director of the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, is an international leader in the development of innovative treatments for neuromuscular diseases. A primary focus of the Center for Gene Therapy is curing muscular dystrophies and Nationwide Children’s Hospital has been part of the Muscular Dystrophy Association’s Clinical Research Network since 2008. Investigators are currently focused on developing treatments for:

  • Amyotrophic lateral sclerosis (ALS)
  • Becker muscular dystrophy
  • Charcot-Marie-Tooth Neuropathy
  • Duchenne muscular dystrophy
  • Fasioscapulohumeral muscular dystrophy
  • Inclusion body myositis
  • Limb girdle muscular dystrophy type 2D (alpha sarcoglycanopathy)
  • Spinal muscular atrophy

Learn about heart research in neuromuscular diseaseduchenne muscular dystrophy research and spinal muscular atrophy research.

Nationwide Children’s has done more gene therapy clinical trials for neuromuscular disease than any place in the world. Right now, our ground-breaking SMA1 clinical trial is providing hope to Tenley and all families affected by SMA.