News

Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...
Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...
Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows ...
Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory ...
What Happened? Shares of biotech company Sarepta Therapeutics (NASDAQ:SRPT) fell 44.4% in the afternoon session after the ...
Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...
A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys ...
Supernus Pharmaceuticals said it is acquiring Sage Therapeutics, the distressed maker of a drug for postpartum depression, ...