News

Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...
Sarepta suspends Elevidys use in non-ambulatory Duchenne patients after two liver failure deaths, triggering FDA review and ...
Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory ...
The drug, Givinostat, has been approved as safe for use but Belfast Health Trust, NI's specialist centre for the condition, ...
Sarepta Therapeutics shares dived 42% in premarket trading after the company disclosed a second reported case of acute liver failure that resulted in a death tied to its Duchenne muscular dystrophy ...
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...
Parents of children with an incurable muscle-wasting condition have called on the Minister for Health to accelerate the ...
Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...
The parents of a nine-year-old boy battling a devastating muscle-wasting condition say new research could offer them more ...
U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support ...