View in EDS HTML Full Text PDF Full Text

A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States.

Saved in:
Bibliographic Details
Title: A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States.
Authors: Pillai NR; Division of Genetics & Metabolism, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA., Solomon F; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Steiner RD; Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, USA., Xie B; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Haselkorn T; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Young C; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Rozario N; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Walzer M; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Schoser B; Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University, Munich, Germany.
Source: Journal of neuromuscular diseases [J Neuromuscul Dis] 2026 May; Vol. 13 (3), pp. 362-372. Date of Electronic Publication: 2025 Oct 28.
Publication Type: Journal Article
Journal Info: Publisher: IOS Press Country of Publication: United States NLM ID: 101649948 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2214-3602 (Electronic) Linking ISSN: 22143599 NLM ISO Abbreviation: J Neuromuscul Dis Subsets: 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: 41148004
AccessLevel: 2
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States.
– Name: Author
  Label: Authors
  Group: Au
  Data: <searchLink fieldCode="AU" term="%22Pillai+NR%22">Pillai NR</searchLink>; Division of Genetics & Metabolism, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.<br /><searchLink fieldCode="AU" term="%22Solomon+F%22">Solomon F</searchLink>; Astellas Pharma Global Development, Inc., Northbrook, IL, USA.<br /><searchLink fieldCode="AU" term="%22Steiner+RD%22">Steiner RD</searchLink>; Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, USA.<br /><searchLink fieldCode="AU" term="%22Xie+B%22">Xie B</searchLink>; Astellas Pharma Global Development, Inc., Northbrook, IL, USA.<br /><searchLink fieldCode="AU" term="%22Haselkorn+T%22">Haselkorn T</searchLink>; Astellas Pharma Global Development, Inc., Northbrook, IL, USA.<br /><searchLink fieldCode="AU" term="%22Young+C%22">Young C</searchLink>; Astellas Pharma Global Development, Inc., Northbrook, IL, USA.<br /><searchLink fieldCode="AU" term="%22Rozario+N%22">Rozario N</searchLink>; Astellas Pharma Global Development, Inc., Northbrook, IL, USA.<br /><searchLink fieldCode="AU" term="%22Walzer+M%22">Walzer M</searchLink>; Astellas Pharma Global Development, Inc., Northbrook, IL, USA.<br /><searchLink fieldCode="AU" term="%22Schoser+B%22">Schoser B</searchLink>; Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University, Munich, Germany.
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%22101649948%22">Journal of neuromuscular diseases</searchLink> [J Neuromuscul Dis] 2026 May; Vol. 13 (3), pp. 362-372. <i>Date of Electronic Publication: </i>2025 Oct 28.
– 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="%22IOS+Press%22">IOS Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101649948 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2214-3602 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2222143599%22">22143599 </searchLink><i>NLM ISO Abbreviation: </i>J Neuromuscul Dis <i>Subsets: </i>MEDLINE
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=41148004
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1177/22143602251387210
    Languages:
      – Code: eng
        Text: English
    PhysicalDescription:
      Pagination:
        StartPage: 362
    Titles:
      – TitleFull: A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Pillai NR
      – PersonEntity:
          Name:
            NameFull: Solomon F
      – PersonEntity:
          Name:
            NameFull: Steiner RD
      – PersonEntity:
          Name:
            NameFull: Xie B
      – PersonEntity:
          Name:
            NameFull: Haselkorn T
      – PersonEntity:
          Name:
            NameFull: Young C
      – PersonEntity:
          Name:
            NameFull: Rozario N
      – PersonEntity:
          Name:
            NameFull: Walzer M
      – PersonEntity:
          Name:
            NameFull: Schoser B
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 01
              M: 05
              Text: 2026 May
              Type: published
              Y: 2026
          Identifiers:
            – Type: issn-electronic
              Value: 2214-3602
          Numbering:
            – Type: volume
              Value: 13
            – Type: issue
              Value: 3
          Titles:
            – TitleFull: Journal of neuromuscular diseases
              Type: main
ResultId 1