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Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II.

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Title: Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II.
Authors: Muenzer J; University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA., Burton BK; Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, Illinois, USA., Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, California, USA., Gutiérrez-Solana LG; Infant Jesus Children's Hospital, Madrid, Spain., Ruiz-Garcia M; National Institute of Pediatrics, Mexico City, Mexico., Jones SA; St Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester, Manchester, UK., Guffon N; Reference Centre for Inherited Metabolic Disorder, Hospices Civils de Lyon, Lyon, France., Inbar-Feigenberg M; University of Toronto, Toronto, Ontario, Canada.; The Hospital for Sick Children, Toronto, Ontario, Canada., Bratkovic D; Women's and Children's Hospital, North Adelaide, South Australia, Australia., Rust S; Manchester University NHS Foundation Trust, Manchester, UK., Hale M; Takeda Development Center Americas, Inc., Cambridge, Massachusetts, USA.; Hale Scientific Statistics, LLC, Beaverton, Oregon, USA., Wu Y; Takeda Development Center Americas, Inc., Lexington, Massachusetts, USA., Yee KS; Takeda Development Center Americas, Inc., Cambridge, Massachusetts, USA., Whiteman DAH; Takeda Development Center Americas, Inc., Lexington, Massachusetts, USA., Alexanderian D; Takeda Development Center Americas, Inc., Lexington, Massachusetts, USA.
Source: Journal of inherited metabolic disease [J Inherit Metab Dis] 2025 May; Vol. 48 (3), pp. e12790. Date of Electronic Publication: 2024 Sep 09.
Publication Type: Clinical Trial, Phase II; Clinical Trial, Phase III; Journal Article; Multicenter Study; Randomized Controlled Trial
Journal Info: Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II.
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  Data: <searchLink fieldCode="AU" term="%22Muenzer+J%22">Muenzer J</searchLink>; University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.<br /><searchLink fieldCode="AU" term="%22Burton+BK%22">Burton BK</searchLink>; Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Harmatz+P%22">Harmatz P</searchLink>; UCSF Benioff Children's Hospital Oakland, Oakland, California, USA.<br /><searchLink fieldCode="AU" term="%22Gutiérrez-Solana+LG%22">Gutiérrez-Solana LG</searchLink>; Infant Jesus Children's Hospital, Madrid, Spain.<br /><searchLink fieldCode="AU" term="%22Ruiz-Garcia+M%22">Ruiz-Garcia M</searchLink>; National Institute of Pediatrics, Mexico City, Mexico.<br /><searchLink fieldCode="AU" term="%22Jones+SA%22">Jones SA</searchLink>; St Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester, Manchester, UK.<br /><searchLink fieldCode="AU" term="%22Guffon+N%22">Guffon N</searchLink>; Reference Centre for Inherited Metabolic Disorder, Hospices Civils de Lyon, Lyon, France.<br /><searchLink fieldCode="AU" term="%22Inbar-Feigenberg+M%22">Inbar-Feigenberg M</searchLink>; University of Toronto, Toronto, Ontario, Canada.; The Hospital for Sick Children, Toronto, Ontario, Canada.<br /><searchLink fieldCode="AU" term="%22Bratkovic+D%22">Bratkovic D</searchLink>; Women's and Children's Hospital, North Adelaide, South Australia, Australia.<br /><searchLink fieldCode="AU" term="%22Rust+S%22">Rust S</searchLink>; Manchester University NHS Foundation Trust, Manchester, UK.<br /><searchLink fieldCode="AU" term="%22Hale+M%22">Hale M</searchLink>; Takeda Development Center Americas, Inc., Cambridge, Massachusetts, USA.; Hale Scientific Statistics, LLC, Beaverton, Oregon, USA.<br /><searchLink fieldCode="AU" term="%22Wu+Y%22">Wu Y</searchLink>; Takeda Development Center Americas, Inc., Lexington, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Yee+KS%22">Yee KS</searchLink>; Takeda Development Center Americas, Inc., Cambridge, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Whiteman+DAH%22">Whiteman DAH</searchLink>; Takeda Development Center Americas, Inc., Lexington, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Alexanderian+D%22">Alexanderian D</searchLink>; Takeda Development Center Americas, Inc., Lexington, Massachusetts, USA.
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  Data: <searchLink fieldCode="JN" term="%227910918%22">Journal of inherited metabolic disease</searchLink> [J Inherit Metab Dis] 2025 May; Vol. 48 (3), pp. e12790. <i>Date of Electronic Publication: </i>2024 Sep 09.
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        Value: 10.1002/jimd.12790
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              Text: 2025 May
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