Fabry disease is a lysomal storage disorder resulting from deficient or absent activity of the lysosomal enzyme, α- galactosidase A, and the resultant accumulation of glycosphingolipids, mainly in the endothelium.

Transthyretin amyloidosis, the most common form of familial amyloidosis, is a systemic disorder characterized by the extracellular deposition of amyloid fibrils causing organ dysfunction and failure.