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

•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 rec...

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Veröffentlicht in:	Molecular genetics and metabolism 2024-09, Vol.143 (1-2), p.108555, Article 108555
Hauptverfasser:	Waisbren, Susan E., Christ, Shawn E., Bilder, Deborah A., Bjoraker, Kendra J., Bolton, Scout, Chamberlin, Sarah, Grant, Mitzie L., Janzen, Darren M., Katz, Rachel, Lubliner, Eugene, Martin, Arianna, McQueen, Kelsey, Moshkovich, Olga, Nguyen-Driver, Mina, Shim, Soo, Stefanatos, Arianna K., Wilkening, Greta, Harding, Cary
Format:	Artikel
Sprache:	eng
Schlagworte:	Child Clinical trials Clinical Trials as Topic Cognition Functional outcomes Humans Neuropsychological outcomes Neuropsychological Tests Phenylketonuria Phenylketonurias - diagnosis PKU
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Beschreibung
Zusammenfassung:	•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.
ISSN:	1096-7192 1096-7206 1096-7206
DOI:	10.1016/j.ymgme.2024.108555