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

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

The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...

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 ...

The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

See what the children of your favourite celebrity couples ... holidays and events with their A List parents, get all the latest pictures, videos and stories from the celebrity kids taking the ...

Eight-year-old Sam Millar has a condition called Duchenne muscular dystrophy, a genetic muscle-wasting disease that progressively gets worse over time. He is a statistical outlier – Duchenne UK says ...

They killed seven people, including three children and a pregnant woman ... Image Clarita Alia, showing the photos of her four sons that were killed by the dreaded Davao Death Squad in Davao ...

In this study, we identified four distinct raw BMI growth trajectory groups grouped by child sex, and the early increase trajectories of boys and girls were between the thresholds for obesity and ...