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

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

Roche discontinued the commercial and clinical use of Elevidys after two cases of fatal acute liver failure.

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

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

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

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

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

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

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