With the advances in molecular genetics and basic sciences during the past decade, a number of novel treatments have been introduced and proven effective in some diseases, such as burosumab, an anti-fibroblast growth factor-23 (FGF23) monoclonal antibody for hypophosphatemic rickets and asfotase alfa, a bone-targeted enzyme replacement therapy for hypophosphatasia. This evidence concerns the gene FGF23 and Dent disease.