Molecular Classification
Enzyme, Glycoside hydrolase
Other Names
Acid maltase, Aglucosidase alfa, Alpha-1,4-glucosidase, Amyloglucosidase, Glucoamylase
Disease Roles
Pompe diseaseGlycogen storage disease type II

Acidic alpha-glucosidase Overview

Acidic alpha-glucosidase is a lysosomal enzyme essential for the degradation of glycogen to glucose within lysosomes. Deficiency or dysfunction leads to Pompe disease, characterized by excessive glycogen accumulation. Recombinant enzyme replacement therapy (alglucosidase alfa) is available.

Mechanism of Action

Enzyme replacement therapy

Biological Functions

Glycogen hydrolysis
Glucose production
Lysosomal degradation

Disease Associations

Pompe disease
Glycogen storage disease type II

Safety Considerations

  • Infusion-related reactions
  • Antibody development
  • Limited efficacy in some patients

Interacting Drugs

Alglucosidase alfa

Associated Biomarkers

Biomarker
Glycogen levels
GAA enzyme activity