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

Duchenne Muscular Dystrophy (DMD) is a rare progressive disorder. People with DMD are missing muscle-protecting protein. This is due to a fault in the gene producing dystrophin. Muscles to become ...

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

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

Duchenne muscular dystrophy (DMD) is a genetic condition that primarily affects people assigned male at birth. However, due to the condition’s X-linked inheritance pattern, only people assigned ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

If you’re living with Duchenne muscular dystrophy (DMD), planning ahead can help support the success of future education, employment, and housing efforts. Duchenne muscular dystrophy (DMD) is a ...

About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a ...