Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

They also started looking for better options. Finally, they volunteered Chase, who's now 8, and Dylan, who's now 5, for a study testing an experimental gene therapy for Duchenne muscular dystrophy.

Neurology > General Neurology Gene Therapy for Duchenne Muscular Dystrophy Gets Expanded Approval — Primary endpoint wasn't met, but other outcomes provided "substantial evidence of ...

After decades of research, gene therapy has been approved for Duchenne Muscular Dystrophy, a degenerative muscle disease diagnosed each year in about 1 in 3,500 newborns, almost all boys.