Intellia’s stock fell 23% after one patient in its Phase III MAGNITUDE trial developed severe liver toxicity that later ...

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.

Entrada has secured the authorisation within the EU Clinical Trial Regulation to begin the Phase I/II trial of ENTR-601-45.

PepGen ends Duchenne drug program after weak Phase 2 results, pivots to DM1 treatment showing early signs of splicing ...

Givinostat can slow the progression of Duchenne muscular dystrophy and is now available to some patients via an early access ...

CureDuchenne, a global nonprofit based in Newport Beach committed to finding and funding a cure for Duchenne muscular ...

Boston-based biotech PepGen Inc. (NASDAQ:PEPG) announced today it will discontinue all Duchenne muscular dystrophy (DMD) ...

Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...