Dublin boy paves the way for new muscle disease treatment | DuchenneXchange

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Dublin boy paves the way for new muscle disease treatment


A Dublin boy has become a pioneer in how doctors treat a common form of Muscular Dystrophy. Jones has Duchenne Muscular Dystrophy, which is the most common kind in children. It typically affects boys, and as he gets older, it is going to get harder to walk and even stand.

Every Friday, Jones and his family make the trip all the way from Dublin to Children’s Healthcare of Atlanta for his treatment. What started out as a trial, has now been approved by the Food and Drug Administration.

Research shows Eteplirsen slows the disease’s progression, allowing children to get stronger and walk for longer. “We now have the first ever approved drug by the FDA, so this is a very exciting and promising time for our patients,” says Pediatric Neurologist Dr. Han Phan.”

 

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