After months of uncertainty and controversy, the FDA has given approval to Sarepta's Duchenne muscular dystrophy drug ... available for the rare genetic muscle-wasting disease in the ...

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

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 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 ...

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 ...

He believed that the underlying molecular defect in Duchenne muscular dystrophy was affected by physical activity which hastened the deterioration of the muscle fibers. On the other hand ...

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

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy ... the protein deficient in the muscle-wasting disease, and ...

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 ...

Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal disorder that results in the loss of muscle function, decline in respiratory and cardiac function and ...