Sarepta Therapeutics is embroiled in a regulatory crisis after the FDA requested a halt on its Duchenne muscular dystrophy gene therapy, Elevidys, following a third patient death, this time linked to a separate investigational limb girdle muscular dystrophy (LGMD) trial using the same viral vector. Sarepta has refused to comply, maintaining shipments to younger ambulatory patients. The situation has raised questions about the safety of AAV-based gene therapies and prompted clinical holds and scrutiny over Sarepta's transparency and communication with regulators.