Sarepta Therapeutics (NASDAQ:SRPT) reported a second fatality from acute liver failure in a patient treated with its gene ...

Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death ...

The commercial and clinical use of Elevidys was discontinued after two fatal cases of acute liver failure.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...

The drug, Givinostat, has been approved as safe for use but Belfast Health Trust, NI's specialist centre for the condition, ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) ...

Parents of children with an incurable muscle-wasting condition have called on the Minister for Health to accelerate the ...

U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support ...

Satellos Bioscience Chief Scientific Officer discusses early-stage trials of the company's Duchenne muscular dystrophy drug, ...

The parents of a nine-year-old boy battling a devastating muscle-wasting condition say new research could offer them more ...

The campaign to commemorate the work of the Muscular Dystrophy Association was created in partnership with agency Yes&.