In a groundbreaking medical achievement, doctors have successfully treated a life-threatening motor neuron disease in a baby ...

The mother took the drug daily for six weeks while the baby herself was given the drug orally, roughly one week after the ...

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 ...

Blood proteins serve as crucial indicators of health and disease risk throughout development. Now, researchers at the ...

There is a catch. If the private equity firms can boost sales of bluebird’s three gene therapies to $600 million—over any ...

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 ...

A toddler was successfully treated for a rare genetic disease, spinal muscular atrophy, after world-first in-womb therapy.