Dataset from A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
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下载链接：
https://doi.org/10.25934/PR00010169
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资源简介：
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.
创建时间：
2025-02-10
