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

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

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

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

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

Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement ...

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

Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the ...

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

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

(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 ...