Gene therapy developer Sarepta Therapeutics has reversed course by temporarily pausing all shipments of Elevidys®, its treatment for Duchenne muscular dystrophy (DMD), following significant regulatory pressure. The FDA requested a voluntary halt amid reports of three patient deaths from acute liver failure linked to Elevidys or related investigational therapies using the same viral vector. Sarepta initially refused to suspend distribution pending further label adjustments but yielded to FDA’s demands to preserve collaboration. The standoff has shaken investor confidence and raised concerns about the safety of adeno-associated virus (AAV)-based gene therapies. Sarepta is working with the FDA to finalize a black box warning and address ongoing safety queries while committed to transparency with patients and the Duchenne community.