The migalastat GLP-HEK assay is the gold standard for determining amenability in patients with Fabry disease

Lignende værker

• The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat
  af: Benjamin, Elfrida R., et al.
  Udgivet: (2017)
• In Vitro and In Vivo Amenability to Migalastat in Fabry Disease
  af: Lenders, Malte, et al.
  Udgivet: (2020)
• 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
  af: Germain, Dominique P., et al.
  Udgivet: (2019)
• Baseline Characteristics of Fabry Disease “Amenable” Migalastat Patients in Argentinian Cohort
  af: Sebastián Jaurretche, et al.
  Udgivet: (2024-01-01)
• Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment
  af: Mauer, Michael, et al.
  Udgivet: (2017)