Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is the most common life-threatening childhood form of muscular dystrophy. As it is caused by genetic defects that lead to missing or non-functional proteins, faculty members are investigating the possibility of gene replacement or gene therapy as treatment options for DMD.

Years of leadership by Nationwide Children’s Hospital faculty in diagnosing and developing new treatment strategies for DMD led the National Institutes of Health to designate Nationwide Children’s Hospital a Center of Research Translation (CORT) designed to accelerate new therapies for muscular dystrophies into clinical practice. 

Nationwide Children's Hospital is also part of the MDA’s Clinical Research Network for Duchenne muscular dystrophy. In 2014, Nationwide Children’s was named the first Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy.

Learn about Heart Research in Neuromuscular Disease.

Learn about research into developing a Newborn Screening for Duchenne Muscular Dystrophy.

Muscular Dystrophy Research Podcast

Hosted by Kevin Flanigan, MD, "This Month in Muscular Dystrophy" highlights the latest in muscular dystrophy and other inherited neuromuscular disease research.

Duchenne Muscular Dystrophy Affects Two Brothers

Jenn McNary's sons both have Duchenne muscular dystrophy. One is receiving experimental therapy and the other is not.

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).