Afegostat is a pharmacological chaperone that specifically and reversibly binds acid-β-glucosidase (GCase) in the endoplasmic reticulum with high affinity.

Afegostat enhances GCase activity in the brain, liver, and spleen. When administered orally at 100 mg/kg for four months to Thy1-aSyn mice, it not only improves motor and nonmotor functions but also eliminates the microglial inflammatory response in the substantia nigra. Additionally, it decreases α-synuclein immunoreactivity within nigral dopaminergic neurons and alters α-synuclein aggregation, reducing small aggregates while increasing large ones.