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

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

One of the demands raised during the protests was access to 'Elevidys', a one-time gene therapy treatment that has the ...

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

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for ...

Some studies of Sarepta Therapeutics' muscular dystrophy drug, Elevidys, have been halted by European regulators after a ...

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

Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for ...

When her son reached three last year, a new name loomed over their lives — Duchenne Muscular Dystrophy (DMD), a ...

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