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Assessment of plasma lyso-Gb(3) for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease

PURPOSE: To assess the utility of globotriaosylsphingosine (lyso-Gb(3)) for clinical monitoring of treatment response in patients with Fabry disease receiving migalastat. METHODS: A post hoc analysis evaluated data from 97 treatment-naive and enzyme replacement therapy (ERT)–experienced patients wit...

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Detalles Bibliográficos
Autores principales:	Bichet, Daniel G., Aerts, Johannes M., Auray-Blais, Christiane, Maruyama, Hiroki, Mehta, Atul B., Skuban, Nina, Krusinska, Eva, Schiffmann, Raphael
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group US 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7790748/ https://www.ncbi.nlm.nih.gov/pubmed/32994552 http://dx.doi.org/10.1038/s41436-020-00968-z

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