Larglustat is used in the Treatment of Gaucher disease (GD)

Gauchers Disease is a sphingolipidoses, a subgroup of lysosomal storage diseases. It is the most common lysosomal storage disease, having an autosomal recessive inheritance and resulting from a deficiency of the glucocerebrosidase enzyme due to a genetic defect on the glucosylceramidase beta (GBA1) gene. The enzyme deficiency results in the inability to break down glucosylceramide in lysosomes, and lipid-laden macrophages accumulate in the tissues.