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, ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

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 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 ...

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 ...

—A new qualitative participatory study explored how powered mobility (PM), in the form of modified ride-on cars, impacts children with spinal muscular atrophy type 1 (SMA1) and their families. Here ...

FRESNO Calif. (KFSN) -- The living room of Zaira De La Cruz's home has been turned into a makeshift hospital room. About seven months after her daughter was born, Zoey was diagnosed with the James ...

Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation index ...

If a boy in New York has been convinced to think he’s a girl, the state Medicaid program has got you covered. But if a boy with muscular dystrophy just wants to walk again, he’s in trouble. That’s the ...