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

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

MDA launches 75th Anniversary campaign and premieres new public service announcement (PSA) to mark 75 years of impact in the ...

Jeremy Bray, who has Type 2 spinal muscular atrophy — a rare degenerative disease that has gradually robbed him of the ability to move his muscles — says he cannot wait for Canada's Drug ...

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 ...