Researchers at the University of Pittsburgh say a new minimally invasive intervention can target the root cause of neural function loss.
An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...
The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...
The US Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for people living with spinal muscular atrophy (SMA).
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