First Gene Therapy for Deadly Form of Muscular Dystrophy Gets FDA Approval for Young Kids

June 22, 2023

(Associated Press) – The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment’s ability to help boys with the inherited disease. The Food and Drug Administration approval provides a new option for some patients with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes weakness, loss of mobility and early death. It almost always affects males. (Read More)