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

Authors :: 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
Source :: Molecular Genetics & Metabolism. Sep2024, Vol. 143 Issue 1/2, pN.PAG-N.PAG. 1p.
Publication Year :: 2024
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. [ABSTRACT FROM AUTHOR]

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