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Orphan drugs

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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
  • Therapeutic area
    • Addiction
    • Allergic conditions
    • Animal health
    • Autoimmune diseases
    • Cardiovascular diseases
    • Central nervous system and neurology
    • Dermatology
    • Endocrinology and metabolic diseases
    • Gastrointestinal diseases
    • Genetic disorders
    • Haematology
    • Hepatology
    • Immunology
    • Infectious diseases
    • Men's health
    • Musculoskeletal diseases
    • Nephrology
    • Oncology
    • Ophthalmology
    • Respiratory diseases
    • Surgery, anaesthesiology and wound care
    • Urology and urogenital diseases
    • Women's health
    • Other project experience
  • Special interest
    • Antibiotics and bacterial infections
    • Biosimilars
    • Medical devices
    • Rare diseases and orphan drugs
    • Vaccines
    • Viral infections and anti-viral treatments
  • Strategic and methodological areas
    • Economic models
    • Endpoint reviews and briefing documents
    • HTA and reimbursement
    • Literature reviews
    • Meta-analyses, indirect comparisons and mixed treatment comparisons
    • Qualitative interview methods
    • Value dossiers
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