Lumizyme

It provides an exogenous source of acid glucosidase (GAA), an essential lysosomal enzyme that is deficient or absent in patients with Pompe disease, leading to intralysosomal glycogen accumulation. Lumizyme consists of the human enzyme GAA, encoded by the most predominant of nine observed haplotypes of the GAA gene. Upon intravenous infusion, carbo... hydrate groups of the Lumizyme molecules bind to mannose-6-phosphate (M6P) receptors on the cell surface. The Lumizyme molecules are taken by receptor-mediated transport into the lysosomes of muscle cells, where they then undergo proteolytic cleavage, increasing their catalytic activity. Similar to the natural human enzyme, Lumizyme degrades glycogen by catalyzing the hydrolysis of -1,4- and -1,6- glycosidic linkages of lysosomal glycogen. Prescribing LumizymeRead More