name: | AlglucosidaseAlfa |
ATC code: | A16AB07 | route: | intravenous |
n-compartments | 2 |
Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme replacement therapy approved for the treatment of Pompe disease (glycogen storage disease type II), a rare genetic disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase. It is administered to help reduce glycogen accumulation in tissues, primarily skeletal and cardiac muscle.
Pharmacokinetics in patients with Pompe disease, both infantile-onset and late-onset, after intravenous infusion. Estimates from published clinical studies in both pediatric and adult populations.