A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme
glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside
accumulates within macrophages leading to cellular engorgement, organomegaly, and organ
system dysfunction. The purpose of this study is to evaluate the safety and efficacy of every
other week dosing of GA-GCB (velaglucerase alfa) in participants with type 1 Gaucher disease
who were previously treated with imiglucerase.

戈谢病（Gaucher disease）是一类罕见的溶酶体贮积症，由葡糖脑苷脂酶（glucocerebrosidase, GCB）缺乏所引发。由于功能性GCB的缺失，葡糖脑苷脂会在巨噬细胞内蓄积，进而导致细胞充盈、器官肿大及器官系统功能障碍。本研究旨在评估每隔一周给药方案下的GA-GCB（维拉苷酶α，velaglucerase alfa）在既往接受过伊米苷酶治疗的1型戈谢病受试者中的安全性与有效性。