Muscle tissue damage appears when muscular dystrophy is induced in a mouse model (middle). But when researchers block the function of two genes that drive unwanted mitochondrial pore formation (right) ...

Some children in Northern Ireland who live with a severe form of muscular dystrophy will be able to access new medication on the health service, BBC News understands. The move from the Belfast Health ...

COLUMBUS, Ohio (WCMH) – It’s being called a pioneering event that holds great promise for children who have Duchenne muscular dystrophy. After decades of research, animal studies, and human trials, ...

Find out if it’s time to ask about your child’s symptoms — and if they could be a sign of Duchenne. Duchenne muscular dystrophy — sometimes referred to simply as Duchenne — is a genetic disorder that ...

His parents Stephen and Jenny have asked Sheffield Children's NHS Foundation Trust to approve the use of drug givinostat, which they hoped would give their son a "relatively normal childhood". Dr Jeff ...

A diagnosis of Duchenne muscular dystrophy (DMD) can come as a shock to parents and caregivers. Resources and support from family and friends can help you and your child navigate the diagnosis and ...

The greatest reduction in skeletal muscle mass was in the lower extremities, a new report shows. Low skeletal muscle mass may be an early manifestation of fabry disease (FD), according to a new report ...

New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated. University of Rochester ...

Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...

A retrospective review of children with neuromuscular disorders and confirmed COVID-19 infection found the vast majority of patients experienced asymptomatic or mild disease, with no reported cases of ...

Givinostat is an oral histone deacetylase (HDAC) inhibitor approved by the FDA in 2024 to treat Duchenne patients ages 6 years and older. Pathologic upregulation of HDAC occurs in DMD, leading to ...

When a doctor tells you that your child has spinal muscular atrophy (SMA), you may feel overwhelmed and full of questions. SMA is rare -- only one in 6,000 to one in 10,000 people have it. So your ...