Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series - 08/09/23
Doi : 10.1016/j.jpeds.2023.113493
Jonathan Gaillard, MD 1, Andrew Ran Gu, MD 2, Erin E. Neil Knierbein, DO 1, ⁎ 
1 Division of Neurology, Department of Pediatrics, University of Michigan, Ann Arbor, MI
2 Department of Pediatrics, University of Michigan, Ann Arbor, MI
∗Reprint requests: Erin E. Neil Knierbein, DO, Division of Neurology, Department of Pediatrics, University of Michigan, Ann Arbor, MI.Division of NeurologyDepartment of PediatricsUniversity of MichiganAnn ArborMI
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.
Le texte complet de cet article est disponible en PDF.Abbreviations : AAV, ALT, AST, DPI, FDA, IV, NEC, NPO, OA, PICC, SMA, SMN, TPN
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© 2023
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Vol 260
Article 113493- septembre 2023 Retour au numéro
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