Every year, thousands of infants in America are diagnosed with devastating genetic diseases through newborn screenings. The ...

He was diagnosed with Duchenne muscular dystrophy at age 5.Family remembers the day everything changed"I can remember the day ...

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

Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and ...

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

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

To date, over 900 Duchenne patients have been treated with delandistrogene moxeparvovec. "The signal for ALF is exceptionally ...

Capricor stock drops as FDA cancels advisory meeting on Duchenne therapy; leadership changes and regulatory tensions fuel uncertainty.

Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain ...

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular ...

Four-year data show preservation of cardiac function, including LVEFSkeletal muscle disease progression continues to slow ...

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