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Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.
Chand, Deepa H; Zaidman, Craig; Arya, Kapil; Millner, Rachel; Farrar, Michelle A; Mackie, Fiona E; Goedeker, Natalie L; Dharnidharka, Vikas R; Dandamudi, Raja; Reyna, Sandra P.
Afiliação
  • Chand DH; Novartis Gene Therapies, Bannockburn, IL; Washington University School of Medicine and St Louis Children's Hospital, St Louis, MO. Electronic address: Deepa.chand@novartis.com.
  • Zaidman C; Washington University School of Medicine and St Louis Children's Hospital, St Louis, MO.
  • Arya K; University of Arkansas for Medical Sciences, Little Rock, AR.
  • Millner R; University of Arkansas for Medical Sciences, Little Rock, AR.
  • Farrar MA; Sydney Children's Hospital Randwick and School of Women's and Children's Health, University of New South Wales, Australia (UNSW) Medicine, UNSW Sydney, Australia.
  • Mackie FE; Sydney Children's Hospital Randwick and School of Women's and Children's Health, University of New South Wales, Australia (UNSW) Medicine, UNSW Sydney, Australia.
  • Goedeker NL; Washington University School of Medicine and St Louis Children's Hospital, St Louis, MO.
  • Dharnidharka VR; Washington University School of Medicine and St Louis Children's Hospital, St Louis, MO.
  • Dandamudi R; Washington University School of Medicine and St Louis Children's Hospital, St Louis, MO.
  • Reyna SP; Novartis Gene Therapies, Bannockburn, IL.
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.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Produtos Biológicos / Proteínas Recombinantes de Fusão / Atrofia Muscular Espinal / Microangiopatias Trombóticas Tipo de estudo: Prognostic_studies Limite: Female / Humans / Infant Idioma: En Revista: J Pediatr Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Produtos Biológicos / Proteínas Recombinantes de Fusão / Atrofia Muscular Espinal / Microangiopatias Trombóticas Tipo de estudo: Prognostic_studies Limite: Female / Humans / Infant Idioma: En Revista: J Pediatr Ano de publicação: 2021 Tipo de documento: Article