Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent ...

Key Takeaways Some studies of Sarepta Therapeutics' muscular dystrophy drug, Elevidys, have been halted by European ...

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...

The Cambridge-based biotech's experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

The European Medicines Agency asked for three clinical trials to be placed on hold until the exact cause of death of a US ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

Patients in Scotland can't get the treatment - which is available in England - despite manufacturers giving it away for free ...

Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the ...

In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...