Muscular Dystrophy Research

Muscular dystrophy is a group of more than 30 genetic (passed down by parents) neuromuscular disorders that are fatal and weaken muscles in the arms, legs, spine, heart and lungs. Each type of muscular dystrophy is passed on by a different gene, but all of the disorders worsen over time.

Researchers at the Center for Gene Therapy believe that gene therapy can treat the underlying cause of muscular dystrophy - something that other drugs and medications can't currently do.  Teams of researchers led by Dr. Jerry Mendell and Dr. Kevin Flanigan perform early phase gene therapy trials at Nationwide Children's Hospital, with the potential to transform treatment for disorders such as Duchenne muscular dystrophy.