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

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

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

(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 ...

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

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

Defeat Duchenne Canada is proud to celebrate its 30th anniversary, marking three decades of relentless progress in the fight ...

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

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

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.