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 ...

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) ...

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, ...

His parents Stephen and Jenny have asked Sheffield Children's NHS Foundation Trust to approve the use of drug givinostat, ...

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 ...

Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy. Researchers ...

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 ...

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 ...

83 percent of children in small study experienced atrophy in at least one muscle group Children with life-threatening respiratory failure who require mechanical ventilation in a pediatric intensive ...

The New York Medicaid program won't cover gene therapies for children who suffer from muscular dystrophy and just want to walk.