ba0006p063 | (1) | ICCBH2017
Imel Erik
, Carpenter Thomas
, Linglart Agnes
, Boot Annemieke
, Hogler Wolfgang
, Padidela Raja
, van't Hoff William
, Portale Anthony
, Mao Meng
, Skrinar Alison
, San Martin Javier
, Whyte Michael P
Objectives: In XLH, musculoskeletal outcomes of current treatment with oral phosphate (Pi)/active vitamin D are suboptimal for many patients. In a Phase 2, open-label study, we tested the hypothesis that KRN23 improves rickets and functional outcomes in XLH children.Methods: Fifty-two children with XLH (ages 512 years at baseline) received KRN23 subcutaneously biweekly (Q2W) or monthly (Q4W). At study entry, most participants had received oral Pi/a...