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 ...

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

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

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 ...

(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 ...

Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...

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

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

Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...

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