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The drug is a combination of calcitriol (the active form of vitamin D) and neutral phosphate salts. Calcitriol acts as a potent agonist of the Vitamin D receptor (VDR), which promotes the intestinal absorption of calcium and phosphate and regulates bone mineralization. In patients with X-linked hypophosphatemia (XLH), mutations in the PHEX gene lead to excessive levels of fibroblast growth factor 23 (FGF23), which causes renal phosphate wasting and inhibits the enzyme 1-alpha-hydroxylase, thereby reducing endogenous calcitriol production. The combination therapy aims to restore serum phosphate levels and improve rickets/osteomalacia. This specific regimen is being evaluated by Peking Union Medical College Hospital in a Phase 4 clinical trial comparing high-dose (60 ng/kg/day) versus low-dose (20 ng/kg/day) calcitriol in pediatric patients aged 1-12.
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