PGN-EDODM1, an experimental DM1 therapy from Pepgen, showed biomarker activity and splicing correction in an early clinical ...

The firm is hoping to outline a potential path to accelerated approval for SGT-003 as it gears up to launch a randomized, controlled Phase III trial.

Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients ...

The update comes as the therapy’s regulatory application awaits a decision from the U.S. Food and Drug Administration later ...

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies ...

Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease biomarker that helps stabilize muscles in patients with limb-girdle muscular ...

The Muscular Dystrophy Association (MDA) concluded its 2026 MDA Clinical & Scientific Conference yesterday, convening over 2,400 attendees from 40 countries to help shape the future of neuromuscular ...

Regenxbio has guided its Duchenne muscular dystrophy (DMD) gene therapy candidate through another test, reporting a clean ...

Sen. Ron Johnson is turning up the heat on the Food and Drug Administration after parents of boys with Duchenne muscular dystrophy told him the agency's recent decisions cut off access to a drug they ...

STELLAR-2, a randomized, double-blind, sham-controlled study, will evaluate the effects of salanersen when initiated ~6 months after onasemnogene abeparvovec-xioi in infants with SMA who received ...

Regenxbio releases new Duchenne trial data showing improved motor function and stable heart measures in early-stage patients.

Solid Biosciences (SLDB) stock dips after new Phase 1/2 trial data for SGT-003, its gene therapy for Duchenne muscular dystrophy. Read more here, ...