An inherited disorder that results from a deficit of an enzyme responsible for breaking down a fatty acid substance within the organelles of cells, called lysosomes.

Fabry disease is a disorder inherited in an x-linked manner, which prevents the body from producing the enzyme responsible for breaking down fat.

Pompe disorder is a genetic disorder caused by the accumulation of complex sugar called glycogen in organs and tissues.

Mucopolysaccharidoses (MPS) are rare diseases related to limited enzyme production - lysosomal alpha-L-iduronidase.

Niemann-Pick Types A and B (NPA and NPB), also called Acid Sphingomyelinase Deficiency (ASMD), are caused by the deficiency of a specific enzyme, acid sphingomyelinase (ASM).

Niemann-Pick Types C (NPC) is a lysosomal lipid storage disease that prevents the body from carrying lipids inside of cells. NPC is caused by mutations in the NPC1 and NPC2 gene.