Sarepta Therapeutics mentioned Sunday that it was halting shipments of its Duchenne muscular dystrophy gene remedy for sufferers who can now not stroll, following the dying of a second one that acquired the remedy.
Sarepta disclosed the primary affected person dying — 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 had been non-ambulatory, that means their illness had progressed to the purpose they relied on a wheelchair. Most youngsters with Duchenne lose the power to stroll by adolescence.
The corporate mentioned early Sunday it was working with consultants to give you an enhanced immunosuppressive routine that would 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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