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

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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Main Authors: Barbara K. Burton, Anne Skalicky, Christoph Baerwald, Deborah A. Bilder, Cary O. Harding, Aaron B. Ilan, Elaina Jurecki, Nicola Longo, David T. Madden, H. Serap Sivri, Gisela Wilcox, Janet Thomas, Kathleen Delaney
Format: article
Language:EN
Published: Elsevier 2021
Subjects:
Phenylketonuria
PKU
Clinical outcome assessments
Adults
Patient-reported outcomes
Observer-reported outcomes
Medicine (General)
R5-920
Biology (General)
QH301-705.5
Online Access:https://doaj.org/article/6a03469383fc40518ebc40fcf2d89078
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https://doaj.org/article/6a03469383fc40518ebc40fcf2d89078

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