Aglucosidase alfa consists of the human enzyme acid alpha-glucosidase (GAA) which is essential for the degradation of glygogen to glucose in lysosomes. It is encoded by the most predominant of nine observed haplotypes of this gene. Aglucosidase alfa is produc…

Avalglucosidase alfa, or NeoGAA, is a drug for enzyme replacement therapy specifically designed for Pompe disease, a rare inherited neuromuscular disorder caused by the deficiency of the alpha-glucosidase (GAA) enzyme. GAA is an essential enzyme that hydrolyz…

Cipaglucosidase alfa (ATB200) is a novel recombinant human acid alpha-glucosidase (GAA) investigated for the treatment of patients with Pompe disease, a rare inherited metabolic disorder characterized by a deficiency in GAA.[A232955] Other types of enzyme rep…