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Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1.
Feldman, Amy G; Parsons, Julie A; Dutmer, Cullen M; Veerapandiyan, Aravindhan; Hafberg, Einar; Maloney, Nolan; Mack, Cara L.
Afiliação
  • Feldman AG; Children's Hospital Colorado, University of Colorado School of Medicine, Denver; Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO. Electronic address: amy.feldman@childrenscolorado.org.
  • Parsons JA; Children's Hospital Colorado, University of Colorado School of Medicine, Denver; Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO.
  • Dutmer CM; Children's Hospital Colorado, University of Colorado School of Medicine, Denver; Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO.
  • Veerapandiyan A; University of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR.
  • Hafberg E; Monroe Carell Jr. Children's Hospital at Vanderbilt, Nashville, TN.
  • Maloney N; University of Colorado School of Medicine, Aurora, CO.
  • Mack CL; Children's Hospital Colorado, University of Colorado School of Medicine, Denver; Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO.
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.
Assuntos

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Terapia Genética / Atrofias Musculares Espinais da Infância / Doença Hepática Induzida por Substâncias e Drogas Limite: Female / Humans / Infant / Male Idioma: En Ano de publicação: 2020 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Terapia Genética / Atrofias Musculares Espinais da Infância / Doença Hepática Induzida por Substâncias e Drogas Limite: Female / Humans / Infant / Male Idioma: En Ano de publicação: 2020 Tipo de documento: Article