Former bodybuilder Tony McCue began stumbling and 'dropping things' two years ago. After being diagnosed with locked-in ...

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

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 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 ...

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.

Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude ...

A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated for ...