X-linked congenital nephrogenic diabetes insipidus (NDI) is a rare inherited disease characterized by various clinical manifestations and finally diagnosed on the basis of the detection of arginine vasopressin receptor type 2 gene (AVPR2) mutations.Though traditional treatment for NDI can partially relieve nonspecific symptoms such as polyuria and polydipsia,it is only a symptomatic treatment with various individual response.In line with the development of gene and pharmacological studies,pharmacological chaperones such as AVP2R antagonist or agonist can dramatically rescue cell-surface expression and function of misfolded AVP2R mutants,thus demonstrate the potential as future therapies for NDI as a result of rescuing misfolded AVP2R mutants.However,its efficacy and safety still needs to be confirmed by further cellular functional and clinical researches.