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

Sarepta Therapeutics on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy for a rare form of muscular ...

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) ...

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

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

A Horsham family is battling to get hold of a drug that will slow the progress of their son's muscular dystrophy.

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

Eric, a beloved brother and cousin, peacefully passed away on May 25, 2025, after bravely facing the challenges of Duchenne ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the deterioration of skeletal muscle in the disease. The use of cardiac ...