Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
Status:
Completed
Trial end date:
2008-01-31
Target enrollment:
Participant gender:
Summary
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 long term safety of enzyme
replacement therapy with DRX008A (VPRIV®, GA-GCB; velaglucerase alfa) in patients with type 1
Gaucher disease.