While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

In year two of the trial, Elevidys demonstrated statistically significant and clinically improvements across three key ...

The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...

A Vancouver family is sending their son on one final journey into the stars, where his ashes will eventually transform into a ...

Joshua Mercieca had a rare condition called Duchenne muscular dystrophy, which is a genetic disorder characterised by ...

Joshua Mercieca defied the odds after he was diagnosed with the muscle-wasting condition Duchenne muscular dystrophy at a ...

The data showed reduced difficulties in standing, walking and running that were statistically significant, the company said.

NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.