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) fell ~38% in the premarket on Monday after the company reported a second fatality from ...

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

Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain patients while it evaluates new safety precautions.

Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for ...

Supernus Pharmaceuticals said it is acquiring Sage Therapeutics, the distressed maker of a drug for postpartum depression, ...

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 Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...

Sarepta halts Elevidys shipments for non-ambulatory DMD patients after a second patient death in three months post gene ...

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