ba0005ht6 | (1) | ECTS2016
Imel Erik
, Carpenter Thomas
, Boot Annemieke
, Hogler Wolfgang
, Linglart Agnes
, Padidela Raja
, van't Hoff William
, Whyte Michael
, Agarwal Sunil
, Chen Chao-Yin
, Skrinar Alison
, Martin Javier San
, Portale Anthony
In XLH, high circulating FGF23 causes hypophosphatemia, rickets, and short stature. In our Phase 2 study, 52 XLH children (ages 5-12 years, ≥Tanner 2) were randomized to receive KRN23 subcutaneously biweekly (Q2W) or monthly (Q4W). Serum phosphate (Pi) was measured biweekly. KRN23 dose was titrated (maximum 2 mg/kg) targeting age-appropriate serum Pi concentrations.The first 36 subjects had a mean 6.6 years of standard-of-care treatment before wash...