Cerezyme® (imiglucerase)
Gaucher disease is an inherited genetic condition that causes fatty deposits to build up in certain organs and bones. People with Gaucher disease do not have enough of the enzyme glucocerebrosidase that helps the body to break down a certain type of fat molecule, glucocerebroside. As a result, cells fill up with the undigested fat. These cells are referred to as Gaucher cells.
The course of the disease is quite variable. The most common symptoms of Gaucher disease are enlargement of the liver and spleen, anemia, reduced platelets (resulting in easy bruising and long clotting times), bone pain, bone infarctions often leading to damage to the shoulder or hip joints, and a generalized demineralization of the bones (osteoporosis). The weakening of the bones can then lead to spontaneous fractures.
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Other Resources:
NORD - National Organization for Rare Diseases
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