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Case Reports
. 2021 Apr:231:265-268.
doi: 10.1016/j.jpeds.2020.11.054. Epub 2020 Nov 28.

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

Deepa H Chand  1 , Craig Zaidman  2 , Kapil Arya  3 , Rachel Millner  3 , Michelle A Farrar  4 , Fiona E Mackie  4 , Natalie L Goedeker  2 , Vikas R Dharnidharka  2 , Raja Dandamudi  2 , Sandra P Reyna  5
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Free article
Case Reports

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

Deepa H Chand et al. J Pediatr. 2021 Apr.
Free article

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.

Keywords: gene therapy; hemolytic uremic syndrome; safety; survival motor neuron.

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