Neuromuscular Disorders Research :: Nationwide Children's Hospital

Neuromuscular Disorders Research

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 Disease, Duchenne Muscular Dystrophy Research and Spinal Muscular Atrophy Research.

Neuromuscular Disorders Research Faculty

Louis Chicoine, MD
Kevin Flanigan, MD
Haiyan Fu, PhD
Scott Harper, PhD
Brian K. Kaspar, PhD
Paul T. Martin, PhD
Douglas M. McCarty, PhD
Jerry R. Mendell, MD
Federica Montanaro, PhD
Louise Rodino-Klapac, PhD
Zarife Sahenk, MD, PhD

featured video

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.

Muscular Dystrophy Association's Clinical Research Network

Nationwide Children’s Hospital is one of five centers to be part of the Muscular Dystrophy Association's Clinical Research Network to support trials and studies in Duchenne muscular dystrophy (DMD).

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