The U.S. Food and Drug Administration has approved the first gene therapy to treat a deadly form of muscular dystrophy.
FILE - A sign for the Food and Drug Administration is displayed outside their offices in Silver Spring, Md., on Dec. 10, 2020. The first gene therapy for Duchenne muscular dystrophy received preliminary U.S. approval on Thursday, June 22, 2023, despite concerns from some government scientists about the treatment's ability to help boys with the inherited disease. – The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S.
The FDA OK'd the treatment only 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. The FDA said the increase in protein seen with the therapy, Elevidys, is “reasonably likely to predict” a benefit in patients 4 to 5 years old, who don’t have other preexisting complications.
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