Imiglucerase

説明Imiglucerase, a mannose-terminated form of human placental glucocerebrosidase produced by recombinant technology, was introduced for the treatment of type-1 Gaucher's disease. lmiglucerase catalyzes the hydrolysis of glucocerebroside and thus prevents accumulation of this lipid in organs and tissues. Compared with the natural enzyme, the modification by removal of the terminal sugars resulted in strong binding of the exposed mannose to lectins expressed on the surface of macrophages, which greatly increases the amount of enzyme taken up by the cells. In 6-month clinical trials, its unequivocal efficacy was shown by improvement in all patients with type-1 Gaucher's disease, leading to an increased hemoglobin levels and platelet counts, decreased incidence of epistaxis and bruising, reduced spleen and liver sizes, and improved skeletal parameters. This enzyme replacement therapy is well tolerated with few mild adverse reactions.

使用Enzyme replenisher (glucocerebrosidase).