Satellos Bioscience's experimental small-molecule drug for Duchenne muscular dystrophy is showing positive results as a stand-alone treatment, but Phil Lambert, Ph.D., the company's chief scientific ...

Repurposing existing therapies may help slow muscle weakness in patients with Duchenne muscular dystrophy, a new study ...

In many genetic diseases, a small error in the DNA code can have devastating effects. These errors, called nonsense mutations ...

Pepgen will stop development of its Duchenne MD exon-skipping therapy PGN-ED051 after increases in dystrophin levels proved ...

"I'm honored to join the Board of Entrada as it advances its clinical-stage portfolio of Duchenne therapies and its growing ...

Dystrophin's isoforms are thought to contribute variably to neuropsychiatric disorders, behavioral disorders, and ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

PepGen (PEPG) announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy patients ...

PepGen to focus on development of DM1 programme following 10 mg/kg PGN-EDO51 update: Boston Friday, May 30, 2025, 14:00 Hrs [IST] PepGen Inc, a clinical-stage biotechnology compan ...

Arrowhead Pharmaceuticals, Inc.'s deal with Sarepta expands its pipeline, but limited plozasiran market and competition ...

CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new DMD gene therapy.