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Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease.
Bichet, Daniel G; Aerts, Johannes M; Auray-Blais, Christiane; Maruyama, Hiroki; Mehta, Atul B; Skuban, Nina; Krusinska, Eva; Schiffmann, Raphael.
Affiliation
  • Bichet DG; Department of Medicine, Hôpital du Sacré Coeur, University of Montréal, Montréal, QC, Canada.
  • Aerts JM; Department of Medical Biochemistry, Leiden Institute of Chemistry, Leiden, Netherlands.
  • Auray-Blais C; Faculty of Medicine and Health Sciences, Université de Sherbrooke, Sherbrooke, QC, Canada.
  • Maruyama H; Department of Clinical Nephroscience, Niigata University Graduate School of Medical and Dental Sciences, Niigata, Japan.
  • Mehta AB; Lysosomal Storage Disorders Unit, University College London, London, UK.
  • Skuban N; Amicus Therapeutics, Cranbury, NJ, USA.
  • Krusinska E; Amicus Therapeutics, Cranbury, NJ, USA.
  • Schiffmann R; Baylor Scott & White Research Institute, Dallas, TX, USA. Raphael.Schiffmann@BSWHealth.org.
Genet Med ; 23(1): 192-201, 2021 01.
Article in En | MEDLINE | ID: mdl-32994552
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Fabry Disease Type of study: Diagnostic_studies / Prognostic_studies Limits: Humans Language: En Journal: Genet Med Journal subject: GENETICA MEDICA Year: 2021 Document type: Article Affiliation country: Canadá Country of publication: Estados Unidos
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Fabry Disease Type of study: Diagnostic_studies / Prognostic_studies Limits: Humans Language: En Journal: Genet Med Journal subject: GENETICA MEDICA Year: 2021 Document type: Article Affiliation country: Canadá Country of publication: Estados Unidos