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. 2023 Oct 27;18(Suppl 2):333. doi: 10.1186/s13023-023-02883-3

Box 5.

Markers of treatment efficacy in XLH

1. Biomarkers (e.g. alkaline phosphatase levels)
2. Clinical manifestations (e.g. lower limb deformities, linear growth)
3. Imaging (e.g. pseudofracture healing, deformity analysis)
4. Patient-reported outcomes (e.g. pain)
These markers depend on the age of the person living with XLH and status of their disease [2, 7, 21]