X-linked hypophosphatemia (XLH)

  • Development of an FDA dossier in X-Linked Hypophosphatemia (XLH)
  • Validation activities of an FDA dossier in XLH
  • Psychometric validation of the Brief Pain Inventory (BPI) and Western Ontario and McMaster osteoarthritis index (WOMAC) in XLH using online survey data
  • Exploration of content validity of PROMIS items for use in children with XLH
  • Psychometric evaluation of the BPI-SF and WOMAC in XLH using phase III trial data