THE NEW ENGLAND JOURNAL OF MEDICINE
Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy
Published February 19, 2025 | DOI: 10.1056/NEJMc2300802 | Copyright © 2025
Abstract
The alternate splicing small molecule risdiplam, administered to a fetus with spinal muscular atrophy, had an apparent ameliorating effect on the disorder through 30 months.
