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 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).

The revolutionary drug, called risdiplam, was taken by the mother during her 32nd week of pregnancy and is now part of the ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...

Spinal muscular atrophy, a progressive neurodegenerative disorder set in motion before birth, could be treated in utero, a ...

This rare genetic disorder was just treated in the womb for the first time ever - Without treatment, those with the more ...

Learn about the promising advance in prenatal treatment of spinal muscular atrophy, introducing a potential approach to suppress symptoms of the disorder in infants.

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

Discover the first in-utero treatment for spinal muscular atrophy (SMA) using risdiplam. Promising results show no SMA signs ...

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...