An infant with a fatal genetic disease has survived past the age of 2 with no signs of the condition, thanks to treatment ...

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

Discover the first in-utero treatment for spinal muscular atrophy (SMA) using risdiplam. Promising results show no SMA signs ...

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

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

Opens in a new tab or window The first prenatal treatment for spinal muscular atrophy showed ... SMA is caused by reduced levels of survival motor neuron (SMN) protein due to deletions or ...

The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more ...

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

February 19, 2025) Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists ...