Gaucher's disease is a lysosomal glycolipid storage disease. The main cause is a gene mutation causing missing of a necessary enzyme. It results in lipid deposition in organs such as bone marrow, liver, spleen, bones and the nervous system. Gaucher's disease can occur at any age. It is the most common in the Central and Eastern European origin (Ashkenazi) Jewish people. Treatment of Gaucher's disease may involve enzyme replacement and other therapies.
How is Gaucher's disease treated in practice?