Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy

Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy

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...