Gaucher Disease Type 1 Clinical Trial
Official title:
Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease
Bone-related problems represent the principal unmet medical need in Gaucher disease (GD). 75% of GD type 1 patients develop skeletal complications, including bone remodeling defects, osteopenia, osteoporosis, marrow infiltration, avascular necrosis, and osteolysis. However, the underlying cellular/molecular basis of bone involvement and related complications in GD are not fully known. Neither are there any bone-specific markers associated with individual bone pathology. Early diagnosis of bone disease is the key issue for planning individual therapy to prevent and reverse bone disease in GD.
This clinical observational study is designed to identify specific biomarkers for bone
involvement in patients with GD1 with decreased bone density and/or bone structural
abnormalities
Aims:
1. Identify novel immune cell surface and biochemical markers in peripheral blood
correlating with bone involvement in GD.
2. Assess the correlation between cytokine levels in peripheral blood and the severity of
bone involvement in GD.
3. Assess the relationship between glycosphingolipids accumulation and macrophage
activation with specific bone markers and GD severity.
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