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).

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 ...

A new study from the University of Ottawa’s Faculty of Medicine sheds light on a potential breakthrough in treating muscle ...

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

Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs ...

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

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