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

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

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 ...

This dystrophin transcript, theoretically, would exist in both muscle and the central nervous system, resulting in Duchenne muscular dystrophy in the former and neuronal dysfunction in the latter ...

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 ...

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 ...

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 ...

Children with a rare muscle wasting disease are being denied ... Givinostat slows the progress of Duchenne muscular dystrophy (DMD) and is being provided free to the NHS by manufacturers after ...

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 ...