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...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:Genet Med
Main Authors: Bichet, Daniel G., Aerts, Johannes M., Auray-Blais, Christiane, Maruyama, Hiroki, Mehta, Atul B., Skuban, Nina, Krusinska, Eva, Schiffmann, Raphael
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group US 2020
Assuntos:
Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7790748/
https://ncbi.nlm.nih.gov/pubmed/32994552
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41436-020-00968-z
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados