In a first for the rare disease, the FDA has approved Sarepta’s gene therapy for Duchenne Muscular Dystrophy, a genetic condition that causes progressive muscle wasting in childhood, primarily affecting boys. The approval covers children between the ages of 4 and 5, contrary to the request from advocacy groups to open up the treatment to all children with the disorder who can still walk.
According to Debra Miller of the patient group CureDuchenne, “Today is a very important day, but every single day these boys are losing muscle cells. And so when you have a son with Duchenne and you see them getting weaker right before your eyes, you know we have to get therapies to patients sooner rather than later.”
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(Source: NPR, June 22nd, 2023)