A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials

INTRODUCTION: Current clinical outcome assessments (COAs) are not effectively capturing the complex array of symptoms of adults with phenylketonuria (PKU). This study aimed to identify concepts of interest relevant to adults with PKU. Based on these concepts, COAs for patient-reported outcomes (PROs...

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Detalles Bibliográficos
Autores principales: Burton, Barbara K., Skalicky, Anne, Baerwald, Christoph, Bilder, Deborah A., Harding, Cary O., Ilan, Aaron B., Jurecki, Elaina, Longo, Nicola, Madden, David T., Sivri, H. Serap, Wilcox, Gisela, Thomas, Janet, Delaney, Kathleen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8591457/
https://www.ncbi.nlm.nih.gov/pubmed/34815941
http://dx.doi.org/10.1016/j.ymgmr.2021.100810

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