DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...

Duchenne muscular dystrophy (DMD ... When blood doesn't pump as well, tachycardia (elevated heart rate) and heart failure (when the heart pumps poorly) can occur. These symptoms may start in ...

its gene therapy for Duchenne muscular dystrophy, the first known death linked to the treatment. The teenager suffered liver failure, a side effect that has been seen with other gene therapy ...

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 ...

"Patients with Duchenne continue to be in need of treatment options that could meaningfully impact the course of disease," said Carolina Tesi-Rocha, M.D., Stanford Children's Hospital. "The ...