Tìm theo
Genz-112638
Thuốc Gốc
Small Molecule
Genz-112638 is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide. It is being developed by the Genzyme Corporation.
Dược Lực Học : Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid β-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a substrate (lipid) called glucosylceramide and, to a lesser degree, glucosphingosine. In patients with Gaucher disease, the liver, spleen, bone marrow and brain show increases in lipid concentration, specifically in cells derived from the monocyte/macrophage system. Genz-112638 is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide.
Cơ Chế Tác Dụng : Genz-112638 is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide. It is being developed by the Genzyme Corporation. Genz-112638, a novel ceramide analog given orally, is designed to inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucocerebroside. This is the substance that builds up in the cells and tissues of people with Gaucher disease. In preclinical studies, the molecule has shown high potency and specificity.
Chỉ Định : Investigated for use/treatment in metabolic disease and neurologic disorders.
Tài Liệu Tham Khảo Thêm
drugbank
http://www.drugbank.ca/drugs/DB05715
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