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Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure.
van der Lee, Johanna H; Morton, Jonathan; Adams, Heather R; Clarke, Lorne; Ebbink, Berendine Johanne; Escolar, Maria L; Giugliani, Roberto; Harmatz, Paul; Hogan, Melissa; Jones, Simon; Kearney, Shauna; Muenzer, Joseph; Rust, Stewart; Semrud-Clikeman, Margaret; Wijburg, Frits A; Yu, Zi-Fan; Janzen, Darren; Shapiro, Elsa.
Afiliação
  • van der Lee JH; Pediatric Clinical Research Office, Emma Children's Hospital, Academic Medical Center, Amsterdam, Netherlands.
  • Morton J; Comradis Limited, Oxford, UK.
  • Adams HR; Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA.
  • Clarke L; British Columbia Children's Hospital Research Institute, University of British Columbia, Vancouver, British Columbia, Canada.
  • Ebbink BJ; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • Escolar ML; Department of Pediatrics, Pittsburgh School of Medicine, Pittsburgh, PA, USA.
  • Giugliani R; Department of Genetics/UFRGS, Medical Genetic Service/HCPA, Porto Alegre, Brazil.
  • Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.
  • Hogan M; Saving Case & Friends, Thompsons Station, TN, USA.
  • Jones S; Willink Biochemical Genetic Unit, Manchester Centre for Genomic Medicine, Saint Mary's Hospital, Manchester, UK.
  • Kearney S; Clinical Paediatric Psychology, Birmingham Children's Hospital NHS Foundation Trust, Birmingham, UK.
  • Muenzer J; Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Rust S; Paediatric Psychosocial Department, Royal Manchester Children's Hospital, Manchester, UK.
  • Semrud-Clikeman M; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
  • Wijburg FA; Department of Pediatrics, Academic Medical Center, Amsterdam, Netherlands.
  • Yu ZF; Statistics Collaborative, Inc., Washington, DC, USA.
  • Janzen D; Institute on Development & Disability, Oregon Health & Science University, Portland, OR, USA.
  • Shapiro E; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA; Shapiro Neuropsychology Consulting, LLC, Portland, OR, USA. Electronic address: shapi004@umn.edu.
Mol Genet Metab ; 121(2): 70-79, 2017 06.
Article em En | MEDLINE | ID: mdl-28501294
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridoses / Cognição Tipo de estudo: Guideline Limite: Child / Humans Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2017 Tipo de documento: Article País de afiliação: Holanda
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Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Mucopolissacaridoses / Cognição Tipo de estudo: Guideline Limite: Child / Humans Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2017 Tipo de documento: Article País de afiliação: Holanda