An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

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 ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

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

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 ...

Each shamrock sold for one dollar will help the Muscular Dystrophy Association help children and teens. "Their life span is to 16 years old, so they are struggling with this disease for so long ...

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ind ...

Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.