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. 2023 Sep:260:113493.
doi: 10.1016/j.jpeds.2023.113493. Epub 2023 May 20.

Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

Jonathan Gaillard  1 , Andrew Ran Gu  2 , Erin E Neil Knierbein  3
Affiliations

Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

Jonathan Gaillard et al. J Pediatr. 2023 Sep.

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.

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Conflict of interest statement

Declaration of Competing Interest The authors declare no conflicts of interest.