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

Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...

Sarepta Therapeutics ( NASDAQ: SRPT) reported a second fatality from acute liver failure in a patient treated with its gene ...

Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory ...

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

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 ...

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

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.

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

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