View in EDS HTML Full Text PDF Full Text

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.

Saved in:
Bibliographic Details
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.
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
Full text is not displayed to guests.
FullText Links:
  – Type: pdflink
Text:
  Availability: 1
Header DbId: mdl
DbLabel: MEDLINE Ultimate
An: 41737901
AccessLevel: 2
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: 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.
– Name: Author
  Label: Authors
  Group: Au
  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 Center for Inherited Metabolic Diseases 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.; Present address: Alexion Pharmaceuticals, Inc., AstraZeneca Rare Disease Boston 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.; Present address: Merck Boston Massachusetts USA.
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%22101568557%22">JIMD reports</searchLink> [JIMD Rep] 2026 Feb 22; Vol. 67 (2), pp. e12443. <i>Date of Electronic Publication: </i>2026 Feb 22 (<i>Print Publication: </i>2026).
– Name: TypePub
  Label: Publication Type
  Group: TypPub
  Data: Journal Article
– Name: TitleSource
  Label: Journal Info
  Group: Src
  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Wiley%22">Wiley </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101568557 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Print <i>ISSN: </i>2192-8304 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2221928304%22">21928304 </searchLink><i>NLM ISO Abbreviation: </i>JIMD Rep <i>Subsets: </i>PubMed not MEDLINE
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=41737901
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1002/jmd2.12443
    Languages:
      – Code: eng
        Text: English
    PhysicalDescription:
      Pagination:
        StartPage: e12443
    Titles:
      – TitleFull: 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.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Muenzer J
      – PersonEntity:
          Name:
            NameFull: Burton BK
      – PersonEntity:
          Name:
            NameFull: Harmatz P
      – PersonEntity:
          Name:
            NameFull: Gutiérrez-Solana LG
      – PersonEntity:
          Name:
            NameFull: Ruiz-Garcia M
      – PersonEntity:
          Name:
            NameFull: Jones SA
      – PersonEntity:
          Name:
            NameFull: Guffon N
      – PersonEntity:
          Name:
            NameFull: Inbar-Feigenberg M
      – PersonEntity:
          Name:
            NameFull: Bratkovic D
      – PersonEntity:
          Name:
            NameFull: Rust S
      – PersonEntity:
          Name:
            NameFull: Hale M
      – PersonEntity:
          Name:
            NameFull: Wu Y
      – PersonEntity:
          Name:
            NameFull: Yee KS
      – PersonEntity:
          Name:
            NameFull: Whiteman DAH
      – PersonEntity:
          Name:
            NameFull: Alexanderian D
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 22
              M: 02
              Text: 2026 Feb 22
              Type: published
              Y: 2026
          Identifiers:
            – Type: issn-print
              Value: 2192-8304
          Numbering:
            – Type: volume
              Value: 67
            – Type: issue
              Value: 2
          Titles:
            – TitleFull: JIMD reports
              Type: main
ResultId 1