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Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
Mendell, Jerry R; Al-Zaidy, Samiah A; Lehman, Kelly J; McColly, Markus; Lowes, Linda P; Alfano, Lindsay N; Reash, Natalie F; Iammarino, Megan A; Church, Kathleen R; Kleyn, Aaron; Meriggioli, Matthew N; Shell, Richard.
Afiliação
  • Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
  • Al-Zaidy SA; Department of Pediatrics, The Ohio State University, Columbus.
  • Lehman KJ; Department of Neurology, The Ohio State University, Columbus.
  • McColly M; Al-Zaidy and Associates, LLC, Columbus, Ohio.
  • Lowes LP; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
  • Alfano LN; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
  • Reash NF; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
  • Iammarino MA; Department of Pediatrics, The Ohio State University, Columbus.
  • Church KR; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
  • Kleyn A; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
  • Meriggioli MN; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
  • Shell R; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio.
JAMA Neurol ; 78(7): 834-841, 2021 07 01.
Article em En | MEDLINE | ID: mdl-33999158
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Produtos Biológicos / Proteínas Recombinantes de Fusão / Atrofias Musculares Espinais da Infância Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Child / Child, preschool / Female / Humans / Male Idioma: En Revista: JAMA Neurol Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Produtos Biológicos / Proteínas Recombinantes de Fusão / Atrofias Musculares Espinais da Infância Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Child / Child, preschool / Female / Humans / Male Idioma: En Revista: JAMA Neurol Ano de publicação: 2021 Tipo de documento: Article