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Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial.
Wijburg, Frits A; Whitley, Chester B; Muenzer, Joseph; Gasperini, Serena; Del Toro, Mireia; Muschol, Nicole; Cleary, Maureen; Sevin, Caroline; Shapiro, Elsa; Bhargava, Parul; Kerr, Douglas; Alexanderian, David.
Afiliación
  • Wijburg FA; Department of Pediatrics, Academic Medical Center, Meibergdreef 9, Amsterdam, the Netherlands.
  • Whitley CB; Gene Therapy Center, University of Minnesota, 420 Delaware St SE, Minneapolis, MN 55455, USA.
  • Muenzer J; Department of Genetics, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599-7487, USA.
  • Gasperini S; Department of Pediatrics, Fondazione MBBM, University of Milano Bicocca, San Gerardo Hospital, Monza, Italy.
  • Del Toro M; Pediatric Neurology Unit, Hospital Universitari Vall D'Hebron, Barcelona, Spain.
  • Muschol N; Department of Pediatrics, University Medical Center Hamburg Eppendorf, Hamburg, Germany.
  • Cleary M; Great Ormond Street Hospital, London WC1N 3JH, UK.
  • Sevin C; Neuropediatrics Unit, GHU Paris-Sud - Hôpital de Bicêtre, Le Kremlin Bicêtre, Paris 94275, France.
  • Shapiro E; Gene Therapy Center, University of Minnesota, 420 Delaware St SE, Minneapolis, MN 55455, USA; Shapiro Neuropsychology Consulting LLC, Portland, OR, USA.
  • Bhargava P; Shire, Lexington, MA, USA.
  • Kerr D; Shire, Lexington, MA, USA.
  • Alexanderian D; Shire, Lexington, MA, USA. Electronic address: dalexanderia@shire.com.
Mol Genet Metab ; 126(2): 121-130, 2019 02.
Article en En | MEDLINE | ID: mdl-30528227
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Sulfatasas / Inyecciones Espinales / Mucopolisacaridosis III Tipo de estudio: Clinical_trials Límite: Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2019 Tipo del documento: Article País de afiliación: Países Bajos
Texto completo
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Sulfatasas / Inyecciones Espinales / Mucopolisacaridosis III Tipo de estudio: Clinical_trials Límite: Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2019 Tipo del documento: Article País de afiliación: Países Bajos