Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec -A Single Centre Experience.

Friese, Johannes; Geitmann, Stephanie; Holzwarth, Dorothea; Müller, Nicole; Sassen, Robert; Baur, Ute; Adler, Kristin; Kirschner, Janbernd

Friese, Johannes; Geitmann, Stephanie; Holzwarth, Dorothea; Müller, Nicole; Sassen, Robert; Baur, Ute; Adler, Kristin; Kirschner, Janbernd.

Afiliação

Friese J; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.
Geitmann S; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.
Holzwarth D; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.
Müller N; Department of Pediatric Cardiology, University Hospital Bonn, Bonn, Germany.
Sassen R; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.
Baur U; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.
Adler K; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.
Kirschner J; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany.

J Neuromuscul Dis ; 8(2): 209-216, 2021.

Article em En | MEDLINE | ID: mdl-33427694

Assuntos

Produtos Biológicos/uso terapêutico; Terapia Genética/métodos; Proteínas Recombinantes de Fusão/uso terapêutico; Atrofias Musculares Espinais da Infância/terapia; Pré-Escolar; Feminino; Alemanha; Humanos; Lactente; Masculino

Palavras-chave

AAV9; Spinal muscular atrophy; adeno-associated viral vector; gene therapy; onasemnogene abeparvovec; safety

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Produtos Biológicos / Proteínas Recombinantes de Fusão / Terapia Genética / Atrofias Musculares Espinais da Infância Limite: Child, preschool / Female / Humans / Infant / Male País/Região como assunto: Europa Idioma: En Ano de publicação: 2021 Tipo de documento: Article

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