Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
- PMID: 37211209
- DOI: 10.1016/j.jpeds.2023.113493
Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
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.
Copyright © 2023 Elsevier Inc. All rights reserved.
Conflict of interest statement
Declaration of Competing Interest The authors declare no conflicts of interest.
Similar articles
-
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9. Lancet Neurol. 2021. PMID: 34536405 Clinical Trial.
-
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.Lancet Neurol. 2021 Apr;20(4):284-293. doi: 10.1016/S1474-4422(21)00001-6. Epub 2021 Mar 17. Lancet Neurol. 2021. PMID: 33743238 Clinical Trial.
-
Safety of Onasemnogene Abeparvovec for Patients With Spinal Muscular Atrophy 8.5 kg or Heavier in a Global Managed Access Program.Pediatr Neurol. 2022 Jul;132:27-32. doi: 10.1016/j.pediatrneurol.2022.05.001. Epub 2022 May 10. Pediatr Neurol. 2022. PMID: 35605311
-
From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1.Pediatr Neurol. 2019 Nov;100:3-11. doi: 10.1016/j.pediatrneurol.2019.06.007. Epub 2019 Jun 13. Pediatr Neurol. 2019. PMID: 31371124 Review.
-
Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy.CNS Drugs. 2022 Sep;36(9):995-1005. doi: 10.1007/s40263-022-00941-1. Epub 2022 Aug 12. CNS Drugs. 2022. PMID: 35960489 Review.
Cited by
-
Extracellular vesicles efficiently deliver survival motor neuron protein to cells in culture.Sci Rep. 2025 Feb 15;15(1):5674. doi: 10.1038/s41598-025-90083-3. Sci Rep. 2025. PMID: 39955442 Free PMC article.
-
A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders.Mol Ther. 2024 Oct 2;32(10):3220-3259. doi: 10.1016/j.ymthe.2024.07.016. Epub 2024 Jul 22. Mol Ther. 2024. PMID: 39044426 Free PMC article.
-
Onasemnogene-abeparvovec administration to premature infants with spinal muscular atrophy.Ann Clin Transl Neurol. 2024 Nov;11(11):3042-3046. doi: 10.1002/acn3.52213. Epub 2024 Sep 28. Ann Clin Transl Neurol. 2024. PMID: 39342433 Free PMC article.
-
Consensus from the Brazilian Academy of Neurology for the diagnosis, genetic counseling, and use of disease-modifying therapies in 5q spinal muscular atrophy.Arq Neuropsiquiatr. 2024 Jan;82(1):1-18. doi: 10.1055/s-0044-1779503. Epub 2024 Feb 5. Arq Neuropsiquiatr. 2024. PMID: 38316428 Free PMC article.
-
Postnatal management of preterm infants with spinal muscular atrophy: experience from German newborn screening.Orphanet J Rare Dis. 2024 Sep 26;19(1):353. doi: 10.1186/s13023-024-03362-z. Orphanet J Rare Dis. 2024. PMID: 39327607 Free PMC article.
MeSH terms
LinkOut - more resources
Full Text Sources
Medical