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Onasemnogene abeparvovec preserves bulbar function in infants with presymptomatic spinal muscular atrophy: a post-hoc analysis of the SPR1NT trial.
Shell, Richard D; McGrattan, Katlyn E; Hurst-Davis, Rebecca; Young, Sally Dunaway; Baranello, Giovanni; Lavrov, Arseniy; O'Brien, Eamonn; Wallach, Shiri; LaMarca, Nicole; Reyna, Sandra P; Darras, Basil T.
Afiliação
  • Shell RD; Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics, The Ohio State University, Columbus, OH, USA. Electronic address: richard.shell@nationwidechildrens.org.
  • McGrattan KE; University of Minnesota, Minneapolis, MN, USA.
  • Hurst-Davis R; Primary Children's Hospital, Salt Lake City, UT, USA.
  • Young SD; Stanford University School of Medicine, Stanford, CA, USA.
  • Baranello G; The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, London, UK; NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Lavrov A; Novartis Gene Therapies, Inc., Cambridge, UK.
  • O'Brien E; Novartis, Dublin, Ireland.
  • Wallach S; Novartis Gene Therapies, Inc., Bannockburn, IL, USA.
  • LaMarca N; Novartis Gene Therapies, Inc., Bannockburn, IL, USA.
  • Reyna SP; Novartis Gene Therapies, Inc., Bannockburn, IL, USA.
  • Darras BT; Boston Children's Hospital, Boston, MA, USA.
Neuromuscul Disord ; 33(8): 670-676, 2023 08.
Article em En | MEDLINE | ID: mdl-37455203
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Transtornos de Deglutição / Atrofias Musculares Espinais da Infância Limite: Child / Humans / Infant Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Transtornos de Deglutição / Atrofias Musculares Espinais da Infância Limite: Child / Humans / Infant Idioma: En Ano de publicação: 2023 Tipo de documento: Article