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3 business days
Shire
Gaucher Disease, Type 1

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of theenzyme 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 treatment protocol is toobserve the safety of velaglucerase alfa in patients with type 1 Gaucher disease who areeither treatment naive (newly diagnosed) or who are currently being treated with theEnzyme Replacement Therapy (ERT) imiglucerase.