METHODS AND COMPOSITIONS RELATED TO CALCIUM HOMEOSTASIS AND FABRY DISEASE

摘要

Methods and compositions are provided for treating arrhythmia in patients with Fabry disease using calcium cycling modulators. Fabry disease is an X-linked lysosomal storage disorder, caused by insufficient activity of a-galactosidase A (a-Gal A). As a result of enzyme deficiency glycosphingolipids (GSLs) with terminal a-D-galactosyl moieties, primarily globotriaosylcerarnide (Gb3), accumulate in multiple organs (Brady, et al., 1967).