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 ...

Charlie has Duchenne muscular dystrophy, a degenerative genetic disease that, until recently, has guaranteed death by early adulthood from cardiac or respiratory failure.

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Delandistrogene moxeparvovec ...

Dr. Jerry Mendell, who co-invented the gene therapy, said "treating boys with Duchenne muscular dystrophy after seeing their natural history and decline over and over and over for the past 50 ...

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.

Cardiomyopathy is the leading cause of death in patients with Duchenne muscular dystrophy, with roughly 90% developing a dilated cardiomyopathy by age 18. 2,3 Cardiomyocyte degeneration results in ...

Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy fell short of its main Phase 3 goal, but the firm contends the full body of evidence supports expanding the therapy’s label to ...

Duchenne muscular dystrophy is rare Duchenne is a rare, progressive disorder in which muscle cells are in a constant state of destruction. Need a break? Play the USA TODAY Daily Crossword Puzzle.

Duchenne Muscular Dystrophy may no longer be a death sentence. Oct. 17, 2012— -- Although he made it to 29 years old with Duchenne muscular dystrophy, Jason Williams was not expected to ...

Deflazacort, a newly FDA-approved drug for Duchenne muscular dystrophy, has an $89,000-a-year price tag. It has been put on hold because of an uproar over drug prices.

By Michelle Castillo February 28, 2013 / 1:35 PM EST / CBS News With an incidence of 1 out of 3,500 male births, Duchenne muscular dystrophy is one of the most common of the orphan or rare diseases.

The Food and Drug Administration Monday approved the first drug to treat Duchenne muscular dystrophy, a rare and lethal muscle weakening disorder that affects about 15,000 Americans.