Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders: safety, tolerability, and pharmacokinetics in healthy subjects

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• 作者：N. Guérard ; O. Morand ; J. Dingemanse
• 关键词：Lucerastat ; Safety ; Tolerability ; Pharmacokinetics
• 刊名：Orphanet Journal of Rare Diseases
• 出版年：2017
• 出版时间：December 2017
• 年：2017
• 卷：12
• 期：1
• 全文大小：543KB
• 刊物主题：Medicine/Public Health, general; Pharmacology/Toxicology; Human Genetics;
• 出版者：BioMed Central
• ISSN：1750-1172
• 卷排序：12

文摘

BackgroundLucerastat, an inhibitor of glucosylceramide synthase, has the potential to restore the balance between synthesis and degradation of glycosphingolipids in glycolipid storage disorders such as Gaucher disease and Fabry disease. The safety, tolerability, and pharmacokinetics of oral lucerastat were evaluated in two separate randomized, double-blind, placebo-controlled, single- and multiple-ascending dose studies (SAD and MAD, respectively) in healthy male subjects.