Journal article
Neurocognitive assessment platform for clinical trials in PKU: White paper developed by the NPKUA neurocognitive workgroup
Molecular genetics and metabolism, v 143(1-2), 108555
01 Sep 2024
PMID: 39089209
Abstract
•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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Details
- Title
- Neurocognitive assessment platform for clinical trials in PKU: White paper developed by the NPKUA neurocognitive workgroup
- Creators
- Susan E. Waisbren - Boston Children's HospitalShawn E. Christ - University of MissouriDeborah A. Bilder - Huntsman (United States)Kendra J. Bjoraker - The Medical Center of AuroraScout Bolton - University of MissouriSarah Chamberlin - Montclair State UniversityMitzie L. Grant - St. Christopher's Hospital for ChildrenDarren M. Janzen - Doernbecher Children's HospitalRachel Katz - Lurie Children's HospitalEugene Lubliner - Hofstra UniversityArianna Martin - Children's Hospital ColoradoKelsey McQueen - National PKU AllianceOlga Moshkovich - Clinical InsightsMina Nguyen-Driver - University of Arkansas for Medical SciencesSoo Shim - Lurie Children's HospitalArianna K. Stefanatos - Children's Hospital of PhiladelphiaGreta Wilkening - Children's Hospital ColoradoCary Harding - Doernbecher Children's Hospital
- Publication Details
- Molecular genetics and metabolism, v 143(1-2), 108555
- Publisher
- Elsevier
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Psychiatry
- Web of Science ID
- WOS:001288525700001
- Other Identifier
- 991022202104004721