The second patient dies after obtaining Duchenne’s gene therapy in Sarepta
Sarepta Therapeutics said on Sunday that it interrupted the expeditions of its gene therapy of Duchenne muscular dystrophy for patients who can no longer walk, after the death of a second person who received the treatment.
Sarepta disclosed the death of the first patient – a 16 -year -old boy – in March. The two occurred from acute liver insufficiency, a side effect that has been observed with other gene therapies. The company said that the two patients were not ambulatory, which means that their disease had progressed to the point that they were counting on a wheelchair. Most children with Duchenne lose the ability to walk in adolescence.
The company said early on Sunday that it worked with experts to offer an improved immunosuppressive diet that could make therapy, called Elenidys, safer for non -ambulatory patients. He declared that he would speak with the Food and Drug Administration of the proposed regime.
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