Many leaders want to help desperate families make a full-court press. Sen. Ron Johnson (R-WI) announced an investigation into ...

Jake Kirchner wanted to play baseball in Belle Plaine. This spring, he is, in a way. Mike Max explains.

Katherine Szarama, who has served as Prasad’s deputy at the Center for Biologics Evaluation and Research since December, joins a long list of temporary leaders at the Department of Health and Human ...

PBGENE-DMD aims to deliver sustained muscle function improvement for DMD patients with mutations between exons 45 and 55.

LUBBOCK, Texas (KCBD) - A Lubbock woman is turning her disability into a super power. Steffni Randale was diagnosed with ...

The Phase 1/2 FUNCTION-DMD study will enroll ambulatory patients and is designed to evaluate safety, tolerability, and efficacy, including dystrophin protein expression and functional outcomes. "The ...

A mother caring for her son with Duchenne muscular dystrophy urges FDA Commissioner Makary to prioritize faster treatment ...

Wave Life Sciences recently released its latest 10-Q report. The company is a clinical-stage biotechnology firm based in ...

During ALS Awareness Month this May, the Muscular Dystrophy Association (MDA) is highlighting unprecedented scientific progress, inspiring personal stories, legislative advocacy, and national ...

Orphan drug designation from the European Commission underscores Solid’s commitment to advance SGT-003 through a global development effort for ...

Jon Anning, a Slippery Rock University professor, has done extensive research on effects and treatment for Duchenne muscular ...

A Santa Cruz family received a fully adaptable van for their 12-year-old son with Duchenne muscular dystrophy, funded by ...