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Treatment is mainly supportive and consists of vitamins, supplemental iron or liver extract to prevent anemia caused by iron deficiency and to alleviate other hematologic problems, blood transfusions for anemia, splenectomy for thrombocytopenia, and strong analgesics for bone pain. Injections of areplacement synthetic enzyme have proven helpful. Gene therapy is an experimental approach. An oral treatment with N-butyl deoxynojirimycin (OGT 918), which inhibits glucocerebrosidase formation, is being evaluated. Clinical trials have shown improvement in the key clinical features of Gaucher’s disease, including liver and spleen size and, to a lesser degree, blood counts.
Source: Professional Guide to Diseases (Eighth Edition), 2005
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