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 ...

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 ...

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 ...

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.