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 ...

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

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

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

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 ...

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

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

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 ...

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