Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients

Autor: Julia Day, Chandrin Jayatilleke, Matthew Roy
Jazyk: angličtina
Rok vydání: 2024
Předmět:
Zdroj: Bone Reports, Vol 23, Iss , Pp 101814- (2024)
Druh dokumentu: article
ISSN: 2352-1872
DOI: 10.1016/j.bonr.2024.101814
Popis: X-linked hypophosphataemia (XLH) is a life-long phosphate-wasting disorder that causes skeletal deformities, pain, stiffness, and fatigue and impairs quality of life. Burosumab was approved for use in adults in 2020. We describe three adults with persistent XLH symptoms who received burosumab treatment in a real-world setting. Patients report improvements in pain, mobility, physical function, energy, fatigue, and mental wellbeing through patient-reported outcome measures, enriched with further detail from written testimonials.
Databáze: Directory of Open Access Journals