Real-world non-interventional post-authorization safety study of long-term use of burosumab in children and adolescents with X-linked hypophosphatemia: first interim analysis

• Main Authors: Annemieke M. Boot (Author), Gema Ariceta (Author), Signe Sparre Beck-Nielsen (Author), Maria Luisa Brandi (Author), Karine Briot (Author), Carmen de Lucas Collantes (Author), Sandro Giannini (Author), Dieter Haffner (Author), Richard Keen (Author), Elena Levtchenko (Author), M. Zulf Mughal (Author), Outi Mӓkitie (Author), Ola Nilsson (Author), Dirk Schnabel (Author), Liana Tripto-Shkolnik (Author), M. Carola Zillikens (Author), Jonathan Liu (Author), Alina Tudor (Author), Francesco Emma (Author)
• Format: Book
• Published: SAGE Publishing, 2024-05-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 patien...