Gaucher Disease Summary

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I found some additional information on the treatment of Gaucher disease. In 2014 a new drug was approved for the treatment of type 1 form of Gaucher disease. The drug is called Cerdelga. It is a gelatin capsule containing eliglustat that can be taken orally. The drug works by slowing the production of fatty material to deposit in the spleen, liver and bone marrow. Studies show that Cerdelga resulted in a greater reduction in spleen volume and improvement in live volume, blood platelet count and red blood cell count. The side effects associated with Cerdelga includes: fatigue, nausea, diarrhea, back pain and upper abdominal pain.

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