抽象的

Velaglucerase alfa in the treatment of Gaucher disease type 1

Thomas A Burrow, Gregory A Grabowski

Gaucher disease is an autosomal recessively inherited lysosomal storage disease that results from the defective activity of the enzyme acid b-glucosidase (glucocerebrosidase). Velaglucerase alfa was recently developed and approved as an alternative form to imiglucerase enzyme therapy. Despite differences in primary structure and glycosylation patterns, recent preclinical and clinical trials of the preparation have shown similar efficacy and safety profiles to those of imiglucerase. The development of alternative therapies, such as velaglucerase alfa for Gaucher disease, is providing clinicians with a larger armamentarium of therapies, allowing for a more personalized approach to patient care