X-LINKED HYPOPHOSPHATAEMIA (XLH)
is a progressive disease characterized by chronic hypophosphataemia due to increased fibroblast growth factor (FGF23) activity, resulting in skeletal defects, muscular dysfunction, and dental abnormalities.1,2
is a progressive disease characterized by chronic hypophosphataemia due to increased fibroblast growth factor (FGF23) activity, resulting in skeletal defects, muscular dysfunction, and dental abnormalities.1,2
XLH can create a significant burden in the daily lives of children.5
Unresolved symptoms in adults can result in disability.6,7
1. Carpenter TO, Imel EA, Holm IA, Jan de Beur SM, Insogna KL. A clinician’s guide to X-linked hypophosphatemia. J Bone Miner Res. 2011;26(7):1381-1388. 2. Ruppe MD. X-Linked Hypophosphatemia. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2017. 3. Gaucher C, Walrant-Debray O, Nguyen TM, Esterle L, Garabedian M, Jehan F. PHEX analysis in 118 pedigrees reveals new genetic clues in hypophosphatemic rickets. Hum Genet. 2009;125(4):401-411. 4. Linglart A, Biosse-Duplan M, Briot K, et al. Therapeutic management of hypophosphatemic rickets from infancy to adulthood. Endocr Connect. 2014;3(1):R13-30. 5. Klepper S. Measures of pediatric function: Child Health Assessment Questionnaire (C-HAQ), Juvenile Arthritis Functional Assessment Scale (JAFAS), Pediatric Outcomes Data Collection Instrument (PODCI), and Activities Scale for Kids (ASK). Arthritis Care Res. 2011;63:S371-S382. doi:10.1002/acr.20635. 6. Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC). American College of Rheumatology Website. https://www.rheumatology.org/I-Am-A/Rheumatologist/Research/Clinician-Researchers/Western-Ontario-McMaster-Universities-Osteoarthritis-Index-WOMAC. Accessed March 1, 2018. 7. Skrinar A, Dvorak-Ewell M, Evins A, et al. The lifelong impact of X-linked hypophosphatemia: results from a burden of disease survey. J Endocr Soc. 2019;3(7):1321-1334.