Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series

Deepa H. Chand, Craig Zaidman, Kapil Arya, Rachel Millner, Michelle A. Farrar, Fiona E. Mackie, Natalie L. Goedeker, Vikas R. Dharnidharka, Raja Dandamudi, Sandra P. Reyna

Research output: Contribution to journalArticlepeer-review

2 Scopus citations

Abstract

Spinal muscular atrophy is treated with onasemnogene abeparvovec, which replaces the missing survival motor neuron 1 gene via an adeno-associated virus vector. As of July 1, 2020, we had identified 3 infants who developed thrombotic microangiopathy following onasemnogene abeparvovec. Early recognition and treatment of drug-induced thrombotic microangiopathy may lessen mortality and morbidity.

Original languageEnglish
Pages (from-to)265-268
Number of pages4
JournalJournal of Pediatrics
Volume231
DOIs
StatePublished - Apr 2021

Keywords

  • gene therapy
  • hemolytic uremic syndrome
  • safety
  • survival motor neuron

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