Sarepta Therapeutics’ gene therapy Elevidys for Duchenne muscular dystrophy (DMD) has been halted amid growing safety concerns and regulatory setbacks both in the U.S. and Europe. After the FDA requested Sarepta to stop shipping Elevidys citing safety issues, Sarepta complied following initial resistance. European regulators also recommended against approval, citing lack of evidence for functional improvement in patients. Families affected by Duchenne face emotional turmoil as treatment access is abruptly suspended. The controversy has negatively impacted Sarepta’s reputation and financial outlook, with Roche, its European marketing partner, also pausing shipments. FDA officials have indicated Sarepta will need new studies to regain market access. This crisis underscores the challenges of accelerated approvals in gene therapies for rare diseases.