Real-world effectiveness of burosumab across age groups: X-linked hypophosphatemia (XLH) Disease Monitoring Program

The Journal of Clinical Endocrinology & Metabolism 2026 Open access

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Authors: L. Ward, Thomas O Carpenter, Hamilton Cassinelli, Pablo Florenzano, Erik A. Imel, Aliya Khan, Ben Johnson, E. K. L. Yang, Marc Vincent, Heather M. Heerssen, Zhiyi Li, Simmons JH
DOI: 10.1210/clinem/dgag140
Cited by: 2
References: 31

Abstract

CONTEXT: X-linked hypophosphatemia (XLH) is a rare disorder characterized by excess fibroblast growth factor 23 (FGF23), leading to chronic hypophosphatemia, osteomalacia, and rickets. OBJECTIVE: To evaluate outcomes up to 3 years following initiation of treatment with anti-FGF23 antibody (burosumab) in a real-world setting among individuals with XLH, stratified by age group, including those excluded from clinical trials (

Fields of Study & Subjects

Parathyroid Disorders and Treatments Magnesium in Health and Disease Occupational and environmental lung diseases

Publication details

Journal / source
The Journal of Clinical Endocrinology & Metabolism
Publisher
The Endocrine Society
Volume
111
Issue
9
Pages
2466–2482
Publication date
3 April 2026
Language
EN