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Target intelligence / Profile preview
Alpha-galactosidase A (GLA)
- Target
- GLA
- Molecular classification
- Enzyme, Glycosidase, Lysosomal enzyme
01
Overview
Alpha-galactosidase A is a lysosomal enzyme that hydrolyzes terminal alpha-galactosyl residues from glycolipids and glycoproteins. It is encoded by the GLA gene. Deficiency leads to Fabry disease, an X-linked lysosomal storage disorder characterized by Gb3 accumulation. Treatments include enzyme replacement therapy and pharmacological chaperone therapy.
- Other names
- GLACeramide trihexosidasealpha-GAL A
02
Mechanism of action
Enzyme replacement, Pharmacological chaperone
03
Biological functions
Glycolipid catabolismGlycoprotein catabolismHydrolysis of alpha-galactosyl residues
04
Disease associations
Fabry diseaseLysosomal storage disorder
05
Safety considerations
Infusion reactions (ERT)Antibody development (ERT)Limited efficacy depending on mutation (Migalastat)
06
Interacting drugs
Migalastat
2 more in the full profile.
07
Biomarkers
Gb3 levelsAlpha-galactosidase A activity in leukocytesGLA gene mutations
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