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

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

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

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

Exercise can make a significant difference for patients with SMA by working alongside other medical advancements to improve ...

A pioneering team at St Jude Children's Research Hospital in the US say they successfully treated a child diagnosed with SMA ...

Mother took Evrysdi for SMA before birth; baby later received Zolgensma. Dr. Richard Finkel at St. Jude led study showing no ...

Finding the right pair of shoes can be a challenge for many, but for Aaron Warren, it was a personal struggle that led to a ...

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

Earnings included compensation expenses related to equity awards. Excluding these special items, adjusted loss per share was 43 cents against a gain of 12 cents per share in the year-ago quarter.