Gene Therapy :: Nationwide Children's Hospital

Gene Therapy

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.

Dr. Jerry Mendell and his team have discovered that gene therapy can treat the underlying cause of muscular dystrophy - something that other drugs and medications can't currently do.

Because of their research, the only early phase gene therapy trials in the world are now in progress at Nationwide Children's Hospital, with the potential to transform treatment for disorders such as Duchenne muscular dystrophy.

See the video below to find out why muscular dystrophy research is unique at Nationwide Children's and why it has earned the hospital a designation as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center.

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