All fetuses affected with disease genes were born. Then, in the case of DMD, for example, if the affected child was a boy, he would mostly likely either die before reproducing or be incapable of ...

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

After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent ...

Parents see symptoms much earlier and some symptoms are already visible when the children are very young. Duchenne Muscular Dystrophy is named after Dr Duchenne de Boulogne, who was one of the ...

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.