Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Status:
Completed
Trial end date:
2013-08-01
Target enrollment:
Participant gender:
Summary
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the
human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme
glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in
the cells of the monocyte-macrophage system.
This is an extension trial to Study NCT00376168 and NCT00712348.