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The main cause of the disease is mutation in the <jats:italic>SMPD1<\/jats:italic> (type A and B) or <jats:italic>NPC1<\/jats:italic> or <jats:italic>NPC2<\/jats:italic> (type C) genes, which lead to the accumulation of lipid substrates in the lysosomes of the liver, brain, spleen, lung, and bone marrow cells. This is followed by multiple cell damage, dysfunction of lysosomes, and finally dysfunction of body organs. So far, about 346, 575, and 30 mutations have been reported in <jats:italic>SMPD1<\/jats:italic>, <jats:italic>NPC1<\/jats:italic>, and <jats:italic>NPC2<\/jats:italic> genes, respectively. Depending on the type of mutation and the clinical symptoms of the disease, the treatment will be different. 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