Sarepta gene therapy deaths highlight tragic rare disease dilemma

US-based Sarepta Therapeutics is in turmoil after revealing that three patients taking its gene therapies have died this year. The deaths drove the US Food and Drug Administration (FDA) to ask Sarepta to halt shipments of Elevidys (delandistrogene moxeparvovec), which targets Duchenne muscular dystrophy (DMD). Other regulators have delayed launches of the drug or denied it altogether.