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 ...

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

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

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 ...

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

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

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 long-term charity supporter from Wigan borough has been recognised in this year’s Kings Birthday Honours List.

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