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A vision for patient-tailored therapy in HPA/PKU with focus on pharmacological chaperones. A) The concept of genotypedependent therapy including some present (and future) alternatives in the treatment of HPA/PKU. ERT, enzyme replacement therapy; GT, gene therapy; AST, antisense therapy. B) Flow chart of translational approach in the process of discovery and validation of pharmacological chaperones. DSF, differential scanning fluorimetry; SPR, surface plasmon resonance.
