J Gaillard et al. J Pediatr 2023; 260: 113493. Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
Background: Onasemnogene abeparvovec treats spinal muscular atrophy by delivering a functional SMN1 gene.
Key finding: This case report documents two cases of medically-treated necrotizing enterocolitis (NEC) that developed shortly after Onasemnogen Abeparvovec which was administered at ~3 weeks of life in two full term infants (born at 40 and 41 weeks).
My take: Consider the diagnosis of NEC in full term infants with SMA who have received gene therapy.
Related blog posts:
- Gene Replacement Therapy for Spinal Muscular Atrophy and Subacute Liver Failure
- Understanding the New Therapies for Spinal Muscular Atrophy



