Contribution of Glucosylsphingosine (Lyso-Gb1) to Treatment Decisions in Patients with Gaucher Disease

Contribution of Glucosylsphingosine (Lyso-Gb1) to Treatment Decisions in Patients with Gaucher Disease

Glucosylsphingosine (lyso-Gb1), the deacylated form of glucocerebroside, was shown to be the most specific and sensitive biomarker for diagnosing Gaucher disease (GD). The aim of this study is to assess the contribution of lyso-Gb1 at the time of diagnosis for treatment decisions in naïve patients w...

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Bibliographic Details
Main Authors: Tama Dinur, Peter Bauer, Christian Beetz, Claudia Cozma, Michal Becker-Cohen, Majdolen Istaiti, Arndt Rolfs, Volha Skrahina, Ari Zimran, Shoshana Revel-Vilk
Format: Article
Language:English
Published: MDPI AG 2023-02-01
Series:International Journal of Molecular Sciences
Subjects:
glucosylsphingosine
lyso-Gb1
Gaucher disease
enzyme replacement therapy (ERT)
substrate reduction therapy (SRT)
Online Access:https://www.mdpi.com/1422-0067/24/4/3945

Internet

https://www.mdpi.com/1422-0067/24/4/3945

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