View in EDS

Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome Survey.

Saved in:
Bibliographic Details
Title: Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome Survey.
Authors: Muenzer J; University of North Carolina at Chapel Hill, Chapel Hill, NC, USA., Botha J; Takeda Pharmaceuticals International AG, Zurich, Switzerland. Electronic address: jaco.botha@takeda.com., Amartino H; Hospital Universitario Austral, Buenos Aires, Argentina., Giugliani R; Department of Genetics/UFRGS, Medical Genetics Service/HCPA, IMASP, DASA Genomica and Casa dos Raros, Porto Alegre, Brazil., Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA., Kampmann C; Johannes Gutenberg University, Mainz, Germany., Link B; University Children's Hospital, Zurich, Switzerland., Lin SP; Mackay Memorial Hospital, Taipei, Taiwan., Molter D; Washington University School of Medicine, St. Louis, MO, USA., Raiman J; Birmingham Children's Hospital, NHS Foundation Trust, UK., Scarpa M; Udine University Hospital, Udine, Italy., Tylki-Szymańska A; Children's Memorial Health Institute, Warsaw, Poland., Jain S; Takeda Development Center Americas, Inc., Cambridge, MA, USA., Whiteman DAH; Takeda Development Center Americas, Inc., Cambridge, MA, USA., Burton BK; Ann & Robert H Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA.
Source: Molecular genetics and metabolism [Mol Genet Metab] 2025 Dec; Vol. 146 (4), pp. 109284. Date of Electronic Publication: 2025 Nov 02.
Publication Type: Journal Article; Multicenter Study; Observational Study
Journal Info: Publisher: Academic Press Country of Publication: United States NLM ID: 9805456 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1096-7206 (Electronic) Linking ISSN: 10967192 NLM ISO Abbreviation: Mol Genet Metab Subsets: MEDLINE
Database: MEDLINE Ultimate
FullText Text:
  Availability: 0
Header DbId: mdl
DbLabel: MEDLINE Ultimate
An: 41232197
AccessLevel: 2
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome Survey.
– 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, NC, USA.<br /><searchLink fieldCode="AU" term="%22Botha+J%22">Botha J</searchLink>; Takeda Pharmaceuticals International AG, Zurich, Switzerland. Electronic address: jaco.botha@takeda.com.<br /><searchLink fieldCode="AU" term="%22Amartino+H%22">Amartino H</searchLink>; Hospital Universitario Austral, Buenos Aires, Argentina.<br /><searchLink fieldCode="AU" term="%22Giugliani+R%22">Giugliani R</searchLink>; Department of Genetics/UFRGS, Medical Genetics Service/HCPA, IMASP, DASA Genomica and Casa dos Raros, Porto Alegre, Brazil.<br /><searchLink fieldCode="AU" term="%22Harmatz+P%22">Harmatz P</searchLink>; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA.<br /><searchLink fieldCode="AU" term="%22Kampmann+C%22">Kampmann C</searchLink>; Johannes Gutenberg University, Mainz, Germany.<br /><searchLink fieldCode="AU" term="%22Link+B%22">Link B</searchLink>; University Children's Hospital, Zurich, Switzerland.<br /><searchLink fieldCode="AU" term="%22Lin+SP%22">Lin SP</searchLink>; Mackay Memorial Hospital, Taipei, Taiwan.<br /><searchLink fieldCode="AU" term="%22Molter+D%22">Molter D</searchLink>; Washington University School of Medicine, St. Louis, MO, USA.<br /><searchLink fieldCode="AU" term="%22Raiman+J%22">Raiman J</searchLink>; Birmingham Children's Hospital, NHS Foundation Trust, UK.<br /><searchLink fieldCode="AU" term="%22Scarpa+M%22">Scarpa M</searchLink>; Udine University Hospital, Udine, Italy.<br /><searchLink fieldCode="AU" term="%22Tylki-Szymańska+A%22">Tylki-Szymańska A</searchLink>; Children's Memorial Health Institute, Warsaw, Poland.<br /><searchLink fieldCode="AU" term="%22Jain+S%22">Jain S</searchLink>; Takeda Development Center Americas, Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Whiteman+DAH%22">Whiteman DAH</searchLink>; Takeda Development Center Americas, Inc., Cambridge, MA, USA.<br /><searchLink fieldCode="AU" term="%22Burton+BK%22">Burton BK</searchLink>; Ann & Robert H Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL, USA.
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%229805456%22">Molecular genetics and metabolism</searchLink> [Mol Genet Metab] 2025 Dec; Vol. 146 (4), pp. 109284. <i>Date of Electronic Publication: </i>2025 Nov 02.
– Name: TypePub
  Label: Publication Type
  Group: TypPub
  Data: Journal Article; Multicenter Study; Observational Study
– Name: TitleSource
  Label: Journal Info
  Group: Src
  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Academic+Press%22">Academic Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>9805456 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1096-7206 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2210967192%22">10967192 </searchLink><i>NLM ISO Abbreviation: </i>Mol Genet Metab <i>Subsets: </i>MEDLINE
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=41232197
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1016/j.ymgme.2025.109284
    Languages:
      – Code: eng
        Text: English
    PhysicalDescription:
      Pagination:
        StartPage: 109284
    Titles:
      – TitleFull: Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome Survey.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Muenzer J
      – PersonEntity:
          Name:
            NameFull: Botha J
      – PersonEntity:
          Name:
            NameFull: Amartino H
      – PersonEntity:
          Name:
            NameFull: Giugliani R
      – PersonEntity:
          Name:
            NameFull: Harmatz P
      – PersonEntity:
          Name:
            NameFull: Kampmann C
      – PersonEntity:
          Name:
            NameFull: Link B
      – PersonEntity:
          Name:
            NameFull: Lin SP
      – PersonEntity:
          Name:
            NameFull: Molter D
      – PersonEntity:
          Name:
            NameFull: Raiman J
      – PersonEntity:
          Name:
            NameFull: Scarpa M
      – PersonEntity:
          Name:
            NameFull: Tylki-Szymańska A
      – PersonEntity:
          Name:
            NameFull: Jain S
      – PersonEntity:
          Name:
            NameFull: Whiteman DAH
      – PersonEntity:
          Name:
            NameFull: Burton BK
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 01
              M: 12
              Text: 2025 Dec
              Type: published
              Y: 2025
          Identifiers:
            – Type: issn-electronic
              Value: 1096-7206
          Numbering:
            – Type: volume
              Value: 146
            – Type: issue
              Value: 4
          Titles:
            – TitleFull: Molecular genetics and metabolism
              Type: main
ResultId 1