Searchable abstracts of presentations at key conferences on calcified tissues

ba0007p204 | (1) | ICCBH2019

A qualitative analysis of the burden-of-illness associated with X-linked hypophosphataemia (XLH) in children and adolescents

Ferizovic Nermina , Mak Catherine , Marshall Jade , Shaw Nick , Mughal Zulf

Objectives: XLH is a rare, genetic, inherited disorder characterised by low blood phosphate which leads to inadequate mineralisation of bone, resulting in a spectrum of skeletal and functional muscle abnormalities, abnormal tooth development, physical and functional impairments. Treatment with conventional therapy places a significant burden on patients and families; it can require complex treatment dosage schedules, is often poorly tolerated, and can be associated with seriou...