@article{aa667075b0a64659b1fe160db8c7e90b,
title = "Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series",
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",
author = "Chand, {Deepa H.} and Craig Zaidman and Kapil Arya and Rachel Millner and Farrar, {Michelle A.} and Mackie, {Fiona E.} and Goedeker, {Natalie L.} and Dharnidharka, {Vikas R.} and Raja Dandamudi and Reyna, {Sandra P.}",
note = "Funding Information: We thank Christina Fleming, PhD, a Novartis Gene Therapies contract employee, and Michael Nissen, ELS, Novartis Gene Therapies, for medical writing and editing support. Publisher Copyright: {\textcopyright} 2020 The Authors",
year = "2021",
month = apr,
doi = "10.1016/j.jpeds.2020.11.054",
language = "English",
volume = "231",
pages = "265--268",
journal = "Journal of Pediatrics",
issn = "0022-3476",
}