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Although the exact molecular mechanisms by which <jats:italic>PHEX<\/jats:italic> mutations cause disturbed phosphate handling in XLH remain unknown, focus for novel therapies has more recently been based upon the finding that the bone\u2010produced phosphaturic hormone fibroblast growth factor\u201023 is elevated in XLH patient plasma. Previous treatment strategies for XLH were based upon phosphate repletion plus active vitamin D analogues, which are difficult to manage, fail to address the primary pathogenesis of the disease, and can have deleterious side effects. A novel therapy for XLH directly targeting fibroblast growth factor\u201023 via a humanized monoclonal antibody (burosumab\u2010twza\/CRYSVITA, henceforth referred to just as burosumab) has emerged as an effective, and recently approved, pharmacological treatment for both children and adults. 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