Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy for a rare muscular dystrophy. Shares of the company plummeted more than 25% ...

Sarepta Therapeutics (SRPT) stock drops 25% as the company reports a patient death linked to Elevidys, its gene therapy developed with Roche (RHHBY). Read more here.

The death of Matthew Rossi has led to calls for new regulations governing school buses transporting special needs students ...

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...

Robert Califf, MD, former commissioner of the FDA, delivered a keynote address at the 2025 Muscular Dystrophy Association ...

While plenty of people are going green in honor of Saint Patrick's Day, one student at East Stroudsburg University is turning ...

Patients with Fuchs endothelial corneal dystrophy with particular genetic markers are at higher risk for surgery and vision loss.

After some earlier data sparked questions from analysts, Avidity Biosciences has released more data for its RNA-based ...

Scholar Rock will present new Phase 3 SAPPHIRE trial data on apitegromab at the 2025 MDA conference, highlighting efficacy ...

MOVE Peds will enroll pediatric FSHD patients across eight clinical sites, capturing both early-onset and later-onset pediatric forms of the disease. Using MRI and functional testing optimized for ...

The East Stroudsburg University campus community is rallying behind a student’s effort to raise funding for muscular dystrophy that was inspired by her bond with a 9-year-old boy battling the ...

A grandmother joined her son and some family friends to abseil 100 feet down from the Falkirk Wheel to raise vital cash for ...