—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 ...
What Is Itvisma, and Why Does It Matter? Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that the FDA has approved to treat spinal muscular atrophy (SMA) in adults, teens, and children 2 or ...
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 ...
Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...
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