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. |
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| 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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| ISSN: | 1573-2665 |
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| DOI: | 10.1002/jimd.12790 |
