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Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease

  • Daniel G. Bichet
  • , Johannes M. Aerts
  • , Christiane Auray-Blais
  • , Hiroki Maruyama
  • , Atul B. Mehta
  • , Nina Skuban
  • , Eva Krusinska
  • , Raphael Schiffmann*
  • *Corresponding author for this work
  • University of Montreal
  • Leiden University
  • Université de Sherbrooke
  • Niigata University
  • University College London
  • Amicus Therapeutics, Cranbury, NJ, USA
  • Baylor Scott & White Health

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Pharmacology, Toxicology and Pharmaceutical Science