Treatment with the drug eliglustat in untreated adults with Gaucher disease type 1 showed significant improvements in liver and spleen size, hemoglobin level, and platelet count. Gaucher disease type 1 is a genetic disease in which there is improper metabolism due to a defect in an enzyme. It is characterized by enlargement of the spleen and liver, anemia, low blood platelets, chronic bone pain, and the failure to grow properly. The disease is a chronic and progressive ...
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