![]() ![]() The company’s one-time IV treatment delivers a replacement gene for the one that is mutated in boys with the condition. The gene therapy had been studied in children up to age 7 by drugmaker Sarepta Therapeutics. 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 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. approval on Thursday despite concerns from some government scientists about the treatment’s ability to help boys with the inherited disease. WASHINGTON (AP) - The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. ![]()
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