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

CENTOGENE GmbH Rostock · 2018
ClinicalTrials.gov · Datasets · License: Public Domain · 2018
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lipid-metabolism
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
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