Medical Design and Outsourcing6h
Study supports minimally invasive neurostimulation for spinal muscle atrophy patients
Researchers at the University of Pittsburgh say a new minimally invasive intervention can target the root cause of neural function loss.
Deadly motor-neuron disease treated in the womb in world 1st
An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...
SMA8h
Child doing well after mother had SMA treatment while pregnant
A mother was given SMA treatment Evrysdi during pregnancy, and her child with SMA, now 2.5, has not shown any signs of the ...
The American Journal of Managed Care9h
Redefining Functional Measures in SMA: Insights From Clinical Research
The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...
Neurology Advisor10h
FDA Approves Tablet Form of Evrysdi for Spinal Muscular Atrophy
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).
Groundbreaking drug treats rare genetic disorder in the womb for first time ever
A two-and-a-half-year-old girl is defying the odds after becoming the first person to be successfully treated for a rare, ...
New Atlas18h
Infant's genetic muscle disorder improved by treating pregnant mother
Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...