An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment started in the womb.

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).

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

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