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First received: October 30, 2023

Clinical Trial: Biomarkers Related to Bone in Pediatric Gaucher Disease Related to Bone Pathology in Pediatric Patients With Gaucher Disease

Study Status: Completed

Recruit Status: RECRUITING

Condition: Gaucher Disease

Study Type: OBSERVATIONAL


Official Title: Pilot Study to Assess Immune Biomarkers and Growth Factors Related to Bone Pathology in Pediatric Patients With Gaucher Disease

Brief Summary: Aims of the observational study is to establish novel blood-based biomarkers for grading bone disease in pediatric patients with Gaucher disease (GD).Patients with clinically confirmed GD: deficient GCase enzyme activity and corresponding genetic analysis will be eligible for enrollment.Levels of Lyso-Gb1, chitotriosidase, and CCL18 will be established for future bone biomarkers correlation analysis.Skeletal involvement will be assessed using standard clinical diagnostic tools, such as skeletal radiology…

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