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).

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

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 ...