Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation ...

Kim Ball received an accessible van from the Muscular Dystrophy Family Foundation. Applications for the next giveaway are ...

Parent Project Muscular Dystrophy (PPMD), the largest U.S. non-profit leading Duchenne and Becker muscular dystrophy care, research, and advocacy efforts, celebrated a powerful and inspiring Walt ...

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

Company on track to report ELEVATE-44-201 data from the first cohort in Q2 2026 and ELEVATE-45-201 data from the first cohort in mid-2026 – -- Expects to initiate global Phase 1/2 MAD clinical study o ...

JACKSON, MI – A Jackson-area firefighter union’s participation in the annual “Fill the Boot” charity drive has once again successfully collected a sizable amount of donations from the community. The ...

The Enquirer and United Way of Greater Cincinnati have joined forces for the 39th year to help families in need with the Wish List program. After wishes are granted, remaining funds assist people with ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...

TEMPE, AZ (AZFamily) — An Arizona State University student is forging a new path of independence, all while raising awareness for those battling neuromuscular disabilities like himself. On Friday ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...