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 ...

Roche announces positive results from EMBARK phase III study of Elevidys in ambulatory individuals with DMD: Basel Tuesday, January 28, 2025, 11:00 Hrs [IST] Roche announced posit ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

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

CONNECT1 has enrolled two cohorts of boys and young men living with DMD amenable to exon 51 skipping ... exon skipping, and muscle tissue concentration. The 10 mg/kg cohort is fully enrolled ...

Percheron Therapeutics says it will abandon its lead drug candidate altogether, following December's Duchenne muscular dystrophy trial flop.

A live webcast will be available in the Investors & Media section of Dyne’s website at https://investors.dyne-tx.com/news-and-events/events-and-presentations and a replay will be accessible for 90 ...

Duchenne muscular dystrophy occurs when that gene fails ... These may include: Muscle biopsy. A small piece of muscle tissue is removed and examined to confirm the diagnosis or rule out another ...