Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1.
J Pediatr
; 225: 252-258.e1, 2020 10.
Article
em En
| MEDLINE
| ID: mdl-32473148
Spinal muscular atrophy is a neurodegenerative disease resulting from irreversible loss of anterior horn cells owing to biallelic deletions/mutations in the survival motor neuron (SMN) 1 gene. Gene replacement therapy using an adeno-associated virus vector containing the SMN gene was approved by the US Food and Drug Administration in May 2019. We report 2 cases of transient, drug-induced liver failure after this therapy.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Oligonucleotídeos
/
Terapia Genética
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Atrofias Musculares Espinais da Infância
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Doença Hepática Induzida por Substâncias e Drogas
Limite:
Female
/
Humans
/
Infant
/
Male
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article