The FDA requires Sarepta Therapeutics to perform new studies to confirm the safety of its Duchenne muscular dystrophy gene therapy Elevidys, after safety concerns led to a voluntary shipment pause. These studies may involve modified dosing or manufacturing changes tested in limited patients rather than full clinical trials. The therapy, which had FDA approval with controversial evidence, has also faced a negative opinion from European regulators, compounding regulatory and financial challenges for Sarepta. Patient families experience distress amid treatment halts and regulatory uncertainty, while Sarepta commits to regulatory channels to resolve labeling and safety issues.