Afegostat is a pharmacological chaperone, which specifically and reversibly binds acid-β-glucosidase (GCase) in the endoplasmic reticulum (ER) with high affinity^[1].

GCase^[1]

Afegostat (AT2101) increases GCase activity in brain, liver, and spleen. Afegostat (100 mg/kg) administrates orally for 4 months to Thy1-aSyn mice improved motor and nonmotor function, abolishes microglial inflammatory response in the substantia nigra, reduces α-synuclein immunoreactivity in nigral dopaminergic neurons, and reduces the number of small α-synuclein aggregates, while increasing the number of large α-synuclein aggregates^[1].

147.17

C₆H₁₃NO₃

169105-89-9

阿戈司他

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