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Mechanisms of pharmacological chaperone or small-molecule activator. (a) A pharmacological chaperone rescues misfolding. In the case of pharmacological chaperones for acid β-glucosidase, the chaperone is competitively replaced by substrate. (b) A small-molecule activator correcting a disordered region allosterically. (c) A small-molecule activator binding in the active site, increasing productive interaction between substrate and catalytic residues.
