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

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

After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent ...

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.

an investigational agent for Duchenne muscular dystrophy; the company has since discontinued the treatment. In 2022, two children with spinal muscular atrophy died of acute liver failure within ...

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

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

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 ...

Having found that in biomedical research, the Bird Rock resident and scientist at La Jolla’s Sanford Burnham Prebys has ...

“The availability of Soliris for children underscores the importance ... supporters who generously give to organizations like Muscular Dystrophy Association because they believe in the power ...

“The availability of Soliris for children underscores the importance of continued ... thanks to donors and supporters who generously give to organizations like Muscular Dystrophy Association because ...