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 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 ...

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 ...

Now approved in the EU, Duvyzat offers an important treatment option for delaying Duchenne muscular dystrophy (DMD) disease ...

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 ...