Neuropsychological endpoints for clinical trials in methylmalonic acidemia and propionic acidemia: A pilot study

Neuropsychological endpoints for clinical trials in methylmalonic acidemia and propionic acidemia: A pilot study

Introduction: This pilot study assessed instruments measuring relatively discrete neuropsychological domains to inform the selection of clinical outcome assessments that may be considered for interventional trials in methylmalonic acidemia (MMA) and propionic acidemia (PA). Methods: Tests and questi...

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Bibliographic Details
Main Authors: Kimberly A. Chapman, Devon MacEachern, Gerald F. Cox, Mavis Waller, Jeanine Fogarty, Suzanne Granger, Miganush Stepanians, Susan Waisbren
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Methylmalonic acidemia
Propionic acidemia
Neuropsychological testing
Cookie theft picture task, Vineland adaptive behavior scale (VABS)
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426922001136

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http://www.sciencedirect.com/science/article/pii/S2214426922001136

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