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

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 inf...

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Veröffentlicht in:The Journal of pediatrics 2023-09, Vol.260, p.113493-113493, Article 113493
Hauptverfasser: Gaillard, Jonathan, Gu, Andrew Ran, Neil Knierbein, Erin E.
Format: Artikel
Sprache:eng
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Zusammenfassung: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.
ISSN:0022-3476
1097-6833
DOI:10.1016/j.jpeds.2023.113493