Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...

Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...

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 ...

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.

The commercial and clinical use of Elevidys was discontinued after two fatal cases of acute liver failure.

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

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

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