Children's Hospital Los Angeles paused Sarepta's Elevidys gene therapy after FDA flagged safety issues, including deaths in non-ambulatory patients.

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still walk. A pause on shipments to older, non-ambulatory patients remains in place.

As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm behind its approved Duchenne muscular dystrophy (DMD) treatment.  | As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head,