Gaucher Disease: Understanding the Role of Enzyme Replacement Therapy

Gaucher disease is a rare inherited lysosomal storage disorder caused by the deficiency of an enzyme called glucocerebrosidase. This enzyme breakdown a fatty substance called glucocerebroside which is mainly found in cells of the liver, spleen, bone marrow, and nervous system. Due to the deficiency of this enzyme, glucocerebroside starts accumulating in these organs and tissues which disrupts their normal functioning.

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