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, ...

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 ...

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 ...

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On March 27, 2024, Ann & Robert H. Lurie Children's Hospital of Chicago treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular ...

This is an archived article and the information in the article may be outdated. Please look at the time stamp on the story to see when it was last updated. INDIANAPOLIS — Officials with the Riley ...

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 ...

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 ...

—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 ...

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 ...