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Deriving Vignettes for the Rare Disease AADC Deficiency Using Parent, Caregiver and Clinician Interviews to Evaluate the Impact on Health-Related Quality of Life

PURPOSE: Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare genetic condition, characterised by movement disorder, and speech and cognitive functioning impairment. To enable economic evaluation of treatments, health-related quality of life or utilities need to be derived. These are curr...

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Dades bibliogràfiques
Publicat a:Patient Relat Outcome Meas
Autors principals: Hanbury, Andria, Smith, Adam B, Buesch, Katharina
Format: Artigo
Idioma:Inglês
Publicat: Dove 2021
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7800699/
https://ncbi.nlm.nih.gov/pubmed/33447119
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/PROM.S278258
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