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

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 ...

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

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

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

JAR of Hope helps support a team of researchers and doctors who are aiming to eliminate Duchenne muscular dystrophy – a fatal ...

Pathologic changes of the X-chromosome gene for dystrophin give rise to Duchenne muscular dystrophy (DMD), and dystrophin -- ...

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

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