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).
Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...
Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs ...
Discover the first in-utero treatment for spinal muscular atrophy (SMA) using risdiplam. Promising results show no SMA signs ...
Doctors say drugs that improve muscle mass could benefit older or infirm people at greater risk of falls and fractures ...
A UK study found more than 80% of 80 SMA type 1 children on a DMT had scoliosis or other abnormal spinal curvature needing ...
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