首页    期刊浏览 2024年11月28日 星期四
登录注册

文章基本信息

  • 标题:Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events
  • 本地全文:下载
  • 作者:Michael Beck ; Derralynn Hughes ; Christoph Kampmann
  • 期刊名称:Molecular Genetics and Metabolism Reports
  • 印刷版ISSN:2214-4269
  • 出版年度:2018
  • 卷号:14
  • 页码:31-35
  • DOI:10.1016/j.ymgmr.2017.10.008
  • 出版社:Elsevier B.V.
  • 摘要:

    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.

  • 关键词:Fabry disease ; Enzyme replacement therapy ; Agalsidase alfa ; Long-term effectiveness
国家哲学社会科学文献中心版权所有