Intrathecal idursulfase-IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single-arm, open-label, phase 2/3 substudy and extension.
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| Title: | Intrathecal idursulfase-IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single-arm, open-label, phase 2/3 substudy and extension. |
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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 Center for Inherited Metabolic Diseases 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.; Present address: Alexion Pharmaceuticals, Inc., AstraZeneca Rare Disease Boston Massachusetts USA., Whiteman DAH; Takeda Development Center Americas, Inc. Lexington Massachusetts USA., Alexanderian D; Takeda Development Center Americas, Inc. Lexington Massachusetts USA.; Present address: Merck Boston Massachusetts USA. |
| Source: | JIMD reports [JIMD Rep] 2026 Feb 22; Vol. 67 (2), pp. e12443. Date of Electronic Publication: 2026 Feb 22 (Print Publication: 2026). |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Wiley Country of Publication: United States NLM ID: 101568557 Publication Model: eCollection Cited Medium: Print ISSN: 2192-8304 (Print) Linking ISSN: 21928304 NLM ISO Abbreviation: JIMD Rep Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 2192-8304 |
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| DOI: | 10.1002/jmd2.12443 |
