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

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

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

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

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

Evrysdi, marketed by Genentech, a Roche company, is a disease-modifying therapy designed to target the genetic root cause of ...

Spinal muscular atrophy (SMA ... SMA is caused by a lack of survival motor neuron protein and occurs in around 1 in every 11,000 births in the United States. If not treated, SMA type 1 (SMA ...

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

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

The FDA on Wednesday approved the industry’s first-ever tablet treatment for spinal muscular atrophy— Roche ’s SMN2 splicing ...