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 ...

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

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

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 ...

What Is Duchenne Muscular Dystrophy? Muscular dystrophies are a group of diseases that make muscles weaker and less flexible over time. Duchenne muscular dystrophy (DMD) is the most common type.

Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness and wasting. It is an X-linked recessive disorder that occurs due to changes in the DMD gene ...

Patients with Duchenne muscular dystrophy now have new hope with gene therapy. And for an Illinois family, it’s a double blessing. Kate Middleton announces her cancer has gone into remission in ...

Satellos has invented SAT-3247 as a first-of-its-kind, orally administered small molecule drug designed to restore skeletal muscle regeneration initially in Duchenne muscular dystrophy (DMD).