Second affected person dies after getting Sarepta’s Duchenne gene remedy
Sarepta Therapeutics mentioned Sunday that it was halting shipments of its Duchenne muscular dystrophy gene remedy for sufferers who can not stroll, following the loss of life of a second one that acquired the therapy.
Sarepta disclosed the primary affected person loss of life — a 16-year-old boy — in March. Each occurred from acute liver failure, a facet impact that has been seen with different gene therapies. The corporate mentioned each sufferers have been non-ambulatory, that means their illness had progressed to the purpose they relied on a wheelchair. Most youngsters with Duchenne lose the flexibility to stroll by adolescence.
The corporate mentioned early Sunday it was working with specialists to give you an enhanced immunosuppressive routine that might make the remedy, known as Elevidys, safer for non-ambulatory sufferers. It mentioned it might speak with the Meals and Drug Administration concerning the the proposed routine.
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