Eliglustat is an oral medication used primarily for the treatment of type 1 Gaucher disease, a rare genetic disorder caused by a deficiency in the enzyme glucocerebrosidase. This deficiency leads to the accumulation of glucocerebroside in various tissues, causing a range of health issues. Eliglustat works by inhibiting the enzyme glucosylceramide synthase, thereby reducing the production of glucocerebroside.
