In a surprising turn of events, the FDA has allowed Sarepta Therapeutics to resume shipments of its gene therapy, Elevidys, for younger Duchenne muscular dystrophy patients who can walk, despite concerns over previous fatalities.
As gene therapy for Duchenne muscular dystrophy faces scrutiny after tragic deaths, families like the Revells navigate the uncertain waters of hope and risk, questioning the future of their loved ones.
Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy is on hold due to safety issues. An FDA official warns the path to market approval is fraught with challenges, especially after patient deaths linked to liver injuries.
Sarepta Therapeutics is under fire as another patient succumbs to acute liver failure linked to its gene therapy trial for muscular dystrophy, adding to the recent deaths of two teenage boys.
