Fabry disease is an X-linked lysosomal storage disorder, caused by mutations in the alpha-galactosidase A (GLA) gene encoding the GLA enzyme.1 Consecutive loss or reduction of GLA activity leads to an accumulation of glycosphingolipids, primarily globotriaosycleramide (Gb3) and its deacylated derivative globotriaosylsphingosine (Lyso-Gb3) in several cell types, including neurons and endothelial cells.2 The gene discussed is GLA; the disease is Fabry disease.