ba0007p74 | (1) | ICCBH2019
Gilbey-Cross Robyn
, Sandy Jessica L
, Morris Mavali
, Cocca Alessandra
, Sakka Sophia D
, Massey Jill
, Cheung Moira S
Objectives: Burosumab, a monoclonal antibody that therapeutically targets the underlying elevated levels of fibroblast growth factor 23 (FGF23) in X-linked hypophosphatemia (XLH), is now available to children out of trial conditions. Our objective was to describe the effect of burosumab on quality of life, functionality and pain in a clinical setting.Methods: Questionnaire tools were completed at baseline, 6 and 9 months for 9 children with XLH starting ...