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 as a Paul. D. Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC). The MDCRC allows Nationwide Children’s Hospital researchers to further develop methods to overcome immune barriers to gene correction for Duchenne muscular dystrophy.
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.
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).
Wellstone Center Designation