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Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial.

著者 Imel EA , Glorieux FH , Whyte MP , Munns CF , Ward LM , Nilsson O , Simmons JH , Padidela R , Namba N , Cheong HI , Pitukcheewanont P , Sochett E , Högler W , Muroya K , Tanaka H , Gottesman GS , Biggin A , Perwad F , Mao M , Chen CY , Skrinar A , San Mar
Lancet.2019 May 16 ; ():.
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X-linked hypophosphataemia in children is characterised by elevated serum concentrations of fibroblast growth factor 23 (FGF23), hypophosphataemia, rickets, lower extremity bowing, and growth impairment. We compared the efficacy and safety of continuing conventional therapy, consisting of oral phosphate and active vitamin D, versus switching to burosumab, a fully human monoclonal antibody against FGF23, in paediatric X-linked hypophosphataemia.
PMID: 31104833 [PubMed - as supplied by publisher]
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