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.
摘要：
Onasemnogeneabeparvovec通过提供功能性SMN1基因治疗脊髓性肌萎缩症（SMA）。坏死性小肠结肠炎（NEC）通常发生在早产儿中。我们报告了两名诊断为SMA的足月婴儿，这些婴儿在输注了asemnogeneabeparvovec后出现了NEC。我们讨论了潜在的病因，并提出了在无基因治疗后监测NEC的建议。
