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Neurocognitive assessment platform for clinical trials in PKU: White paper developed by the NPKUA neurocognitive workgroup
Journal article   Peer reviewed

Neurocognitive assessment platform for clinical trials in PKU: White paper developed by the NPKUA neurocognitive workgroup

Susan E. Waisbren, Shawn E. Christ, Deborah A. Bilder, Kendra J. Bjoraker, Scout Bolton, Sarah Chamberlin, Mitzie L. Grant, Darren M. Janzen, Rachel Katz, Eugene Lubliner, …
Molecular genetics and metabolism, v 143(1-2), 108555
01 Sep 2024
DOI: https://doi.org/10.1016/j.ymgme.2024.108555
PMID: 39089209
Featured in Collection :   UN Sustainable Development Goals @ Drexel

Abstract

Functional outcomes Neuropsychological outcomes Phenylketonuria PKU Clinical Trials
•This White Paper offers a neurocognitive assessment platform for clinical trials in PKU.•It describes 5 neuropsychological domains relevant to outcomes in PKU.•Criteria for measures to be included in the Assessment Platform are defined.•Both Performance and Patient Reported Outcome measures are recommended.•Once implemented, this Platform may accelerate approval of new PKU treatments.

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UN Sustainable Development Goals (SDGs)

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#3 Good Health and Well-Being

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Collaboration types
Domestic collaboration
Web of Science research areas
Endocrinology & Metabolism
Genetics & Heredity
Medicine, Research & Experimental
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