—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 ...
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 ...
Darren Ormiston, a 9-year-old boy with Duchenne muscular dystrophy, suddenly screamed and lept from his wheelchair, running to a doctor with his hands in the air. Darren wasn’t in distress, just ...
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 ...
A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study ...
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