Target intelligence / Profile preview

Acidic alpha-glucosidase (GAA)

Target: GAA
Molecular classification: Enzyme, Glycoside hydrolase

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.

Other names: Acid maltaseAglucosidase alfaAlpha-1,4-glucosidaseAmyloglucosidaseGlucoamylase

Mechanism of action

Enzyme replacement therapy

Biological functions

Glycogen hydrolysisGlucose productionLysosomal degradation

Disease associations

Pompe diseaseGlycogen storage disease type II

Safety considerations

Infusion-related reactionsAntibody developmentLimited efficacy in some patients

Interacting drugs

Alglucosidase alfa

Biomarkers

Glycogen levelsGAA enzyme activity

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