脊髓性肌萎缩症 Onasemnogene Abeparvovec 治疗后发生坏死性小肠结肠炎：病例系列。
Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.
机构信息
Division of Neurology, Department of Pediatrics, University of Michigan, Ann Arbor, MI.
Department of Pediatrics, University of Michigan, Ann Arbor, MI.
出版信息
J Pediatr. 2023 Sep;260:113493. doi: 10.1016/j.jpeds.2023.113493. Epub 2023 May 20.
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.
Onasemnogene abeparvovec 通过递送功能性 SMN1 基因治疗脊髓性肌萎缩症。坏死性小肠结肠炎通常发生于早产儿。我们报告了 2 例接受 Onasemnogene abeparvovec 输注后发生坏死性小肠结肠炎的足月婴儿脊髓性肌萎缩症病例。我们讨论了潜在的病因，并提出在 Onasemnogene abeparvovec 治疗后监测坏死性小肠结肠炎。
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