Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events

Michael Beck, Derralynn Hughes, Christoph Kampmann, Guillem Pintos-Morell, Uma Ramaswami, Michael L. West, Roberto Giugliani

Research output: Contribution to journalArticleResearchpeer-review

6 Citations (Scopus)

Abstract

© 2017 The Authors This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately. Cardiac events occurred at younger ages than cerebrovascular or renal events, cerebrovascular events were more frequent in females than males, and males were more likely to experience cardiac and renal events at a younger age than females.
Original languageEnglish
Pages (from-to)31-35
JournalMolecular Genetics and Metabolism Reports
Volume14
DOIs
Publication statusPublished - 1 Mar 2018

Keywords

  • Agalsidase alfa
  • Enzyme replacement therapy
  • Fabry disease
  • Long-term effectiveness

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