Gene therapy has emerged as a promising approach to previously untreatable conditions, including Duchenne muscular dystrophy (DMD), a progressive disorder caused by DMD mutations that leads to early ...

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 ...

Health Secretary Neil Gray is facing a furious backlash after failing to respond to an urgent plea to help children with muscular dystrophy ... boys with Duchenne who are losing muscle every ...

Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada The Canadian regulatory submission of vamorolone ...

Here are the causes and treatment for the congenital disorder Duchenne muscular dystrophy! Entering the age of 2 years, almost most children will start to actively explore their surroundings. However, ...

Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada The Canadian regulatory submission of vamorolone ...

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 ...