Annemieke M Boot, Gema Ariceta, Signe Sparre Beck-Nielsen, Maria Luisa Brandi, Karine Briot, Carmen de Lucas Collantes, Sandro Giannini, Dieter Haffner, Richard Keen, Elena Levtchenko, M Zulf Mughal, Outi Makitie, Ola Nilsson, Dirk Schnabel, Liana Tripto-Shkolnik, M Carola Zillikens, Jonathan Liu, Alina Tudor, Francesco Emma
BACKGROUND: X-linked hypophosphatemia (XLH) is a rare, progressive disorder characterized by excess fibroblast growth factor 23 (FGF23), causing renal phosphate-wasting and impaired active vitamin D synthesis. Burosumab is a recombinant human monoclonal antibody that inhibits FGF23, restoring patient serum phosphate levels. Safety data on long-term burosumab treatment are currently limited. OBJECTIVES: This post-authorization safety study (PASS) aims to monitor long-term safety outcomes in children and adolescents (1-17 years) treated with burosumab for XLH...
2024: Therapeutic Advances in Chronic Disease