Glucosylsphingosine (Lyso-Gb1) as a reliable biomarker in Gaucher disease: a narrative review.

Gaetano GiuffridaUros MarkovicAnnalisa CondorelliValeria CalafioreDaniela NicolosiMarianna CalagnaStephanie GrassoMarco Tindaro Valentino RagusaJennifer GentileMariasanta Napolitano

Published in: Orphanet journal of rare diseases (2023)

Lyso-Gb1 is the most promising biomarker of GD, as demonstrated by its reliability in reflecting disease burden and monitoring treatment response. Furthermore, lyso-Gb1 may play an important role in the onset of monoclonal gammopathy of uncertain significance, multiple myeloma, and Parkinson's disease in GD patients.

Keyphrases

multiple myeloma
newly diagnosed
ejection fraction
prognostic factors
risk factors
patient reported