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 ...

Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients with baseline cardiomyopathy (p=0.017)Global Statistical ...

MDA Clinical & Scientific Conference The 2026 MDA Clinical & Scientific Conference Highlights Breakthroughs in Neuromuscular Research, Innovative Therapies, and Patient Care Orlando, Florida, March 12 ...

BridgeBio Pharma Inc. BBIO on Wednesday announced a recent presentation of interim results from its Phase 3 FORTIFY trial for BBP-418, a treatment for limb-girdle muscular dystrophy type 2I/R9.

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 ...

SGT-003 in the INSPIRE DUCHENNE trial to date - - Robust microdystrophin expression with consistent mechanistic evidence of dystrophin associated protein complex (DAPC) restoration and improvements in ...

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, ...

Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases.