Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

Authors
Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2018
International, multicenter, epidemiological study to demonstrate the correlation and predictive value of lyso-Gb1 concentration with the clinical severity of naïve, initially non-ERT/SRT Gaucher disease type 1 and during the study ERT/SRT-newly started Gaucher type 1 patients and to correlate lyso-Gb1 concentration with the clinical improvement of ERT or SRT treated Gaucher type 1 and the clinical course of non-treated patients based on GD-DS3
Epistemonikos ID: 667916e5856f5b143ddd4c655505fcdc5b21b147
First added on: May 12, 2024