Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

著录项

• 来源
  《Genetics in medicine》 |2019年第9期|共11页
• 作者

  Germain Dominique P.; Nicholls Kathy; Giugliani Roberto; Bichet Daniel G.; Hughes Derralynn A.; Barisoni Laura M.; Colvin Robert B.; Jennette J. Charles; Skuban Nina; Castelli Jeffrey P.; Benjamin Elfrida; Barth Jay A.; Viereck Christopher;

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• 作者单位

  Paris Saclay Univ Univ Versailles Div Med Genet Montigny France;

  Univ Melbourne Royal Melbourne Hosp Parkville Vic Australia;

  Univ Fed Rio Grande do Sul HCPA Med Genet Serv Porto Alegre RS Brazil;

  Univ Montreal Hop Sacre Coeur Dept Nephrol Montreal PQ Canada;

  Royal Free NHS Fdn Trust London England;

  Univ Miami Miller Sch Med Dept Pathol Miami FL 33136 USA;

  Harvard Med Sch Massachusetts Gen Hosp Boston MA 02115 USA;

  Univ North Carolina Chapel Hill Sch Med Chapel Hill NC USA;

  Amicus Therapeut Inc Cranbury NJ USA;

  Amicus Therapeut Inc Cranbury NJ USA;

  Amicus Therapeut Inc Cranbury NJ USA;

  Amicus Therapeut Inc Cranbury NJ USA;

  Amicus Therapeut Inc Cranbury NJ USA;

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• 收录信息
• 原文格式 PDF
• 正文语种 eng
• 中图分类 医学遗传学;
• 关键词

  Fabry disease; migalastat; classic; pharmacogenetics; precision medicine;

  机译：法布里疾病;Migalastat;经典;药物发生;精密药物;