Researchers at the University of Pittsburgh say a new minimally invasive intervention can target the root cause of neural function loss.

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 ...

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).

Neurofilament light (NFL) is a powerful and sensitive biomarker for tracking neuronal health and disease progression.

A two-and-a-half-year-old girl is defying the odds after becoming the first person to be successfully treated for a rare, ...

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 ...

Spinal muscular atrophy is a genetic condition set in motion before birth that causes worsening muscle weakness. There are ...

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