The Muscular Dystrophy Association (MDA) is honoring a longtime researcher and an LGMD patient advocate with its 2025 Legacy ...

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

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

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

The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did ...

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

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

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

Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.