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Start Over Author "Lubliner, Eugene" Topic functional outcomes
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1. Neurocognitive assessment platform for clinical trials in PKU: White paper developed by the NPKUA neurocognitive workgroup.

Author

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, and Harding, Cary

Subjects
*PHENYLKETONURIA, *CLINICAL trials, *FUNCTIONAL status
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]

Published
2024
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