Evaluation of Lyso-Gb1 as a biomarker for Gaucher disease treatment outcomes using data from the Gaucher Outcome Survey

Abstract Background Patients with Gaucher disease (GD) require continual monitoring; however, lack of specific disease biomarkers was a significant challenge in the past. Glucosylsphingosine (lyso-Gb1) has been shown to be a reliable, key, specific, and sensitive biomarker for diagnosis, prognosis,...

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Bibliographic Details
Published in:	Orphanet Journal of Rare Diseases
Main Authors:	Ari Zimran, Shoshana Revel-Vilk, Tama Dinur, Majdolen Istaiti, Jaco Botha, Elena Lukina, Pilar Giraldo, Patrick Deegan, Stephan vom Dahl
Format:	Article
Language:	English
Published:	BMC 2025-01-01
Subjects:	Lyso-Gb1 Glucosylsphingosine Gaucher disease Biomarker
Online Access:	https://doi.org/10.1186/s13023-024-03444-y

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