What is Duchenne Muscular Dystrophy: [ Ссылка ]
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“When they signed up for this they didn't know what the outcome would be but now they have to feel this great sense of gratification for their participation. They have helped basically thousands of boys with Duchenne Muscular Dystrophy.” - Jerry Mendell, MD

Easton and Connor. Both names will go down in history as pioneers for Duchenne Muscular Dystrophy. After being diagnosed with DMD, both boys participated in a novel gene therapy developed and tested at Nationwide Children's Hospital. Not knowing the outcome, not knowing what the future would hold they ultimately paved a path for other kids with DMD.

An extraordinary achievement also for Dr. Mendell. Searching for a cure for nearly 50 years, he has dedicated his life to gene therapy research. Now, with full FDA approval, other boys like Connor and Easton have hope.

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