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

A 12-year-old boy with a severe form of muscular dystrophy ... Duchenne UK, said: "It's just hard to understand with a disease that is so cruel and progressive, and time is muscle, why children ...

The two brothers have Duchenne muscular dystrophy — a rare, inherited muscle-wasting disorder that has no cure. "Caleb stopped walking in December of '23, so a little over a year ago.

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

Duchenne muscular dystrophy (DMD) is a genetic condition ... Each time a person who is a carrier of DMD has a child who is assigned male at birth, the child has a 50% chance of developing DMD.