The CHMP recommends conditional marketing authorization for Duvyzat to treat Duchenne muscular dystrophy in patients aged 6 ...

A new study led by researchers at Hospital for Special Surgery (HSS) suggests that currently available therapies may help ...

A new study led by researchers at the Hospital for Special Surgery (HSS) suggests that currently available therapies may help ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory boys with Duchenne muscular dystrophy, interim data show.“Several microdyst ...

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the latest treatment advancements for Duchenne muscular dystrophy.

Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health care system hundreds of thousands in misdiagnoses, unnecessary treatments and ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

The major reasons behind Duchenne muscular dystrophy include mutations that consist of internal deletions within the gene that codes for dystrophin, a protein that plays a critical role in the ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD ... It causes relentless muscle breakdown, loss of ...

Suneel Ram’s needs gradually became greater after he was diagnosed at age 3 with Duchenne muscular dystrophy, a muscle-wasting genetic disorder that afflicts one in 5,000 boys. Today ...