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.
Orphan drug designation from the European Commission underscores Solid’s commitment to advance SGT-003 through a global ...
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 ...
Nxera Pharma (“the Company” or “Nxera”; TSE: 4565) provides an update on operational activities and reports its consolidated ...
He is a fighter who refuses to let his illness take away his passion for gaming. Despite a progressive muscular disease, ...
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 ...