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

CONCLUSION: Existing COAs relevant to adults with PKU were selected and PKU-specific global impression items were developed by mapping the most frequently identified concepts of interests from internationally-conducted in-depth interviews. Future studies should address the appropriateness of the selected COAs and global impression items to assess if these can be used as efficacy endpoints in PKU clinical trials.

via https://pubmed.ncbi.nlm.nih.gov/34815941/?utm_source=no_user_agent&utm_medium=rss&utm_campaign=None&utm_content=1HYeX0emtvYgH07Wkz0a8n9otrdMd-JIklc_uo0I5vh1u9WMEy&fc=None&ff=20211125010803&v=2.15.0

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