Mutations that cause spinal muscular atrophy (SMA) may lead to abnormalities in the development of cells in the spinal cord, a study found.

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 ...

Add Yahoo as a preferred source to see more of our stories on Google. Biogen said the trial touted the once-yearly dosing potential of the ASO. Credit: Tada Images / Shutterstock.com (Tada Images / ...

SMA newborn screening may help infants start treatment sooner, which researchers say could support better motor development outcomes.

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 ...

On June 2, the Reds caught the attention of Sports Business Journal, Yahoo News and other national media with an action-packed night of baseball and music at Great American Ball Park, punctuated by a ...

The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal ...

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...