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

Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...

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 ...

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

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

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 ...

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

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

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

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