First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids
The HinduThe 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. Still, the FDA's outside experts voted narrowly in favor of making the gene therapy available on a preliminary basis, noting the deadly nature of Duchenne and the risk of delaying a potentially beneficial treatment. The FDA advisers who backed the drug The gene therapy was the latest treatment OK'd through the FDA's fast-track route, which allows drugs to launch based on early results, before they’re confirmed to benefit patients. That means patients who receive Sarepta's gene therapy might be ineligible for future treatments that use viruses, FDA staff said.
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