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

Autor:	Kapil Arya, Deepa H. Chand, Vikas R. Dharnidharka, Michelle A. Farrar, Sandra P. Reyna, Natalie L. Goedeker, Raja Dandamudi, Fiona E. Mackie, Craig M. Zaidman, Rachel Millner
Rok vydání:	2021
Předmět:	Biological Products Pathology medicine.medical_specialty Thrombotic microangiopathy Thrombotic Microangiopathies business.industry Recombinant Fusion Proteins Genetic enhancement Infant Spinal muscular atrophy Motor neuron medicine.disease Viral vector Muscular Atrophy Spinal 03 medical and health sciences 0302 clinical medicine medicine.anatomical_structure 030225 pediatrics Pediatrics Perinatology and Child Health medicine Humans Female 030212 general & internal medicine business
Zdroj:	The Journal of Pediatrics. 231:265-268
ISSN:	0022-3476
Popis:	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.
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_dedup___::66a127dffdaff1a0becd20a845571963 https://doi.org/10.1016/j.jpeds.2020.11.054 Zobrazit plný text záznamu Full Text from ScienceDirect