Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.
J Pediatr
; 231: 265-268, 2021 04.
Article
em En
| MEDLINE
| ID: mdl-33259859
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.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Produtos Biológicos
/
Proteínas Recombinantes de Fusão
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Atrofia Muscular Espinal
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Microangiopatias Trombóticas
Tipo de estudo:
Prognostic_studies
Limite:
Female
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Humans
/
Infant
Idioma:
En
Revista:
J Pediatr
Ano de publicação:
2021
Tipo de documento:
Article