Insights into the Value of Lyso-Gb1 as a Predictive Biomarker in Treatment-Naïve Patients with Gaucher Disease Type 1 in the LYSO-PROOF Study

Insights into the Value of Lyso-Gb1 as a Predictive Biomarker in Treatment-Naïve Patients with Gaucher Disease Type 1 in the LYSO-PROOF Study

Gaucher disease (GD) is a rare autosomal recessive disorder arising from bi-allelic variants in the <i>GBA1</i> gene, encoding glucocerebrosidase. Deficiency of this enzyme leads to progressive accumulation of the sphingolipid glucosylsphingosine (lyso-Gb1). The international, multicente...

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Bibliographic Details
Main Authors: Filipa Curado, Sabine Rösner, Susanne Zielke, Gina Westphal, Ulrike Grittner, Volha Skrahina, Mohammed Alasel, Ahmad Mehmood Malik, Christian Beetz, Tobias Böttcher, Gal Barel, Ashish Prasad Sah, Tama Dinur, Nadeem Anjum, Quidad Ichraf, Yamna Kriouile, Zahra Hadipour, Fatemeh Hadipour, Shoshana Revel-Vilk, Claudia Cozma, Jörg Hartkamp, Huma Cheema, Ari Zimran, Peter Bauer, Arndt Rolfs
Format: Article
Language:English
Published: MDPI AG 2023-08-01
Series:Diagnostics
Subjects:
Gaucher disease
lyso-Gb1
glucosylsphingosine
biomarkers
GD-DS3
Online Access:https://www.mdpi.com/2075-4418/13/17/2812

Internet

https://www.mdpi.com/2075-4418/13/17/2812

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