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

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 ...

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 ...

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

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

Santhera Pharmaceuticals’ Agamree (vamorolone) has been accepted by the Scottish Medicines Consortium (SMC) to treat Duchenne ...

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

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). DMD, a severe genetic disorder that causes muscle weakness and shortens lifespans, arises from mutations in ...