Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort. Delandistrogene ...

Delandistrogene moxeparvovec-rokl shows significant motor function improvements in 8- and 9-year-olds with DMD, countering expected motor decline. Statistically significant differences were observed ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...

Duchenne muscular dystrophy (DMD) occurs as a result of genetic changes on the X chromosome. If someone has a gene change that can cause DMD, their children may inherit that change. DMD is a ...