First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids
The U.S. Food and Drug Administration has approved the first gene therapy to treat a deadly form of muscular dystrophy
WASHINGTON (AP) — 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.
The FDA granted approval for children ages 4 and 5, based on study results showing the therapy helped produce a protein needed for muscle growth, which is missing in boys with the condition. The gene therapy had been studied in children up to age 7 by drugmaker Sarepta Therapeutics.
The company's one-time IV treatment delivers a replacement gene for the one that is mutated in boys with the condition.
