Enzyme replacement therapy. This approach replaces the deficient enzyme with synthetic enzymes. These replacement enzymes are administered in an outpatient procedure through a vein (intravenously), typically at two-week intervals at high doses.
Although effectiveness can vary, it's frequently effective in people with type 1 Gaucher's disease and, in some cases, type 3. In many people, enzyme replacement therapy can reduce the enlargement of the liver and spleen, and help to resolve blood abnormalities. Children treated with enzyme therapy often experience a growth spurt and weight gain as skeletal abnormalities are moderated.
This treatment is most effective when started prior to development of significant organ enlargement or bone impairment. It's unclear whether this therapy is effective for the neurological problems of Gaucher's disease. Occasionally, people experience an allergic or hypersensitivity reaction to enzyme treatment, triggering a rash, nausea, diarrhea or abdominal pain.