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Therapy development for the mucopolysaccharidoses: Updated consensus recommendations for neuropsychological endpoints.
van der Lee, Johanna H; Morton, Jonathan; Adams, Heather R; Clarke, Lorne; Eisengart, Julie B; Escolar, Maria L; Giugliani, Roberto; Harmatz, Paul; Hogan, Melissa; Kearney, Shauna; Muenzer, Joseph; Muschol, Nicole; Rust, Stewart; Saville, Benjamin R; Semrud-Clikeman, Margaret; Wang, Raymond; Shapiro, Elsa.
Affiliation
  • van der Lee JH; Knowledge Institute of the Dutch Association of Medical Specialists, Utrecht, Netherlands; Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Pediatric Clinical Research Office, Amsterdam, Netherlands.
  • Morton J; Comradis, Oxford, UK.
  • Adams HR; Department of Neurology, University of Rochester Medical Center, Rochester, NY, USA.
  • Clarke L; Department of Medical Genetics, Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada.
  • Eisengart JB; Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
  • Escolar ML; Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
  • Giugliani R; Department of Genetics, UFRGS, and Medical Genetics Service, HPCA, Porto Alegre, Brazil.
  • Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.
  • Hogan M; Doulots LLC, Thompson Station, TN, USA.
  • 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.
  • Muschol N; Department of Pediatric, University Medical Center Hamburg Eppendorf, Hamburg, Germany.
  • Rust S; Paediatric Psychosocial Department, Royal Manchester Children's Hospital, Manchester, UK.
  • Saville BR; Berry Consultants LLC, Austin, TX, USA; Vanderbilt University School of Medicine, Nashville, TN, USA.
  • Semrud-Clikeman M; Department of Medical Genetics, Child and Family Research Institute, University of British Columbia, Vancouver, BC, Canada.
  • Wang R; Division of Metabolic Disorders, Children's Hospital of Orange County, Orange, CA, USA.
  • Shapiro E; Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; Shapiro Neuropsychology Consulting LLC, Portland, OR, USA. Electronic address: shapi004@umn.edu.
Mol Genet Metab ; 131(1-2): 181-196, 2020.
Article in En | MEDLINE | ID: mdl-32917509
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Brain / Physical Therapy Modalities / Mucopolysaccharidoses / Nervous System Diseases Type of study: Guideline Aspects: Patient_preference Limits: Humans Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2020 Document type: Article Affiliation country: Países Bajos Country of publication: Estados Unidos
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Brain / Physical Therapy Modalities / Mucopolysaccharidoses / Nervous System Diseases Type of study: Guideline Aspects: Patient_preference Limits: Humans Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2020 Document type: Article Affiliation country: Países Bajos Country of publication: Estados Unidos