Post-authorisation safety study of burosumab use in paediatric, adolescent and adult patients with X-linked hypophosphataemia: rationale and description

• Main Authors: Maria Luisa Brandi (Author), Gema Ariceta (Author), Signe Sparre Beck-Nielsen (Author), Annemieke M. Boot (Author), Karine Briot (Author), Carmen de Lucas Collantes (Author), Francesco Emma (Author), Sandro Giannini (Author), Dieter Haffner (Author), Richard Keen (Author), Elena Levtchenko (Author), Outi Mӓkitie (Author), Ola Nilsson (Author), Dirk Schnabel (Author), Liana Tripto-Shkolnik (Author), M. Carola Zillikens (Author), Jonathan Liu (Author), Alina Tudor (Author), M. Zulf Mughal (Author)
• Format: Book
• Published: SAGE Publishing, 2022-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Background: X-linked hypophosphataemia (XLH) is a rare, inherited, phosphate-wasting disorder that elevates fibroblast growth factor 23 (FGF23), causing renal phosphate-wasting and impaired active vitamin D (1,25(OH) 2 D) synthesis. Disease characteristics include rickets, osteomalacia, odontomalaci...