Related Experiment Video
Updated: Jul 29, 2025

05:55
Delivery of Therapeutic Agents Through Intracerebroventricular ICV and Intravenous IV Injection in Mice
Published on: October 3, 2011
61.0K
Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
Jonathan Gaillard1, Andrew Ran Gu2, Erin E Neil Knierbein1
1Division of Neurology, Department of Pediatrics, University of Michigan, Ann Arbor, MI.
The Journal of Pediatrics
|May 21, 2023
Abstract:
Onasemnogene abeparvovec treats spinal muscular atrophy by delivering a functional SMN1 gene. Necrotizing enterocolitis typically occurs in preterm infants. We report 2 term infants diagnosed with spinal muscular atrophy who presented with necrotizing enterocolitis after onasemnogene abeparvovec infusion. We discuss potential etiologies and propose monitoring for necrotizing enterocolitis after onasemnogene abeparvovec therapy.

