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 ...

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

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 ...

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 new role of calcium in the development of muscles has been reported by two RIKEN biologists.

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