This year's Futures National Conference, hosted by CureDuchenne for the Duchenne and Becker MD communities, is scheduled to ...

Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the importance of a smooth transition from pediatric to adult care for patients with ...

Michael Rankin, 12, who is desperately waiting for access to the drug, said: “Every second they don’t get this through, one ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

A trial found magnetic resonance imaging correlated with physical function tests in patients with Duchenne muscular dystrophy ...

When actor Eric Dane revealed his diagnosis of amyotrophic lateral sclerosis (ALS), the world took notice. Novant Health ...

Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health ...

Sarepta Therapeutics SRPT announced updates from its clinical programs, which focus on targeting different subtypes of ...

Pratteln, Switzerland, April 16, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the latest progress on the commercial rollout of AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD).

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced ...