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Home | Central nervous system | Aromatic L-amino acid decarboxylase (AADC) deficiency | Publications | Publication: Deriving vignettes for the rare disease AADC deficiency using parent, caregiver and clinician interviews to evaluate the impact on health-related quality of life

Publication: Deriving vignettes for the rare disease AADC deficiency using parent, caregiver and clinician interviews to evaluate the impact on health-related quality of life

This research article, published in Journal of Patient-Reported Outcomes, describes the 5-stage development of vignettes describing aromatic L-amino acid decarboxylase (AADC), for use in a subsequent health-state utility elicitation study

  • Learn about the process of development of a symptoms matrix and draft vignettes describing AADC deficiency motor milestones
  • Understand how hypothetical health-state vignettes can subsequently be used to develop health-state utilities for AADC deficiency
  • Recognize the potential importance of health-state utilities in capturing what it is like to live with AADC deficiency, and their utility in economic modelling to investigate the cost-effectiveness of AADC deficiency treatments

Hanbury A, et al. Patient Relat Outcome Meas. 2021;12:1–12

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MED-ALL-AADC-2200023 | June 2025

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