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

A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys ...

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

Sarepta Therapeutics ( NASDAQ: SRPT) fell ~38% in the premarket on Monday after the company reported a second fatality from ...

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 ...

A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile. | A second patient has ...

Shares in both companies were down following news of the second death, casting fresh safety concerns over Elevidys.

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

Sarepta suspends Elevidys use in non-ambulatory Duchenne patients after two liver failure deaths, triggering FDA review and ...

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

Sarepta Therapeutics shares sank to their lowest level in nine years Monday after a second patient taking its Elevidys drug died.