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 ...

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.

Dystrophin's isoforms are thought to contribute variably to neuropsychiatric disorders, behavioral disorders, and ...

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 ...

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

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

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 ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...