Abstract
Excess FGF23 causes hypophosphatemia, leading to chronic debilitating musculoskeletal impairments in patients with X-linked hypophosphatemia (XLH). Treatments include burosumab, a fully-human monoclonal antibody to FGF23, or the combination of oral phosphate and active vitamin D (Pi/D). XLH symptoms in adults include bone/joint pain, stiffness, and fatigue. Previous research validated the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) in adults with XLH. This study assesses burosumab impact on WOMAC among adults from an XLH Disease Monitoring Program (XLH-DMP; NCT03651505).
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