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 ...

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

(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 ...

Donald Trump continues to make waves in biopharma; Sage rejects Biogen’s unsolicited takeover offer; the obesity space sees ...

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

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

An unprecedented number of donations mean this year’s Northamptonshire Jigsaw Festival is set to be the best one yet.

PepGen (PEPG) “provided recent updates on its CONNECT clinical program investigating PGN-EDO51 in Duchenne muscular dystrophy for patients ...

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

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