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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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Wedi'i Gadw mewn:
Prif Awduron: Kimberly A. Chapman, Devon MacEachern, Gerald F. Cox, Mavis Waller, Jeanine Fogarty, Suzanne Granger, Miganush Stepanians, Susan Waisbren
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Elsevier 2023-03-01
Cyfres:Molecular Genetics and Metabolism Reports
Pynciau:
Methylmalonic acidemia
Propionic acidemia
Neuropsychological testing
Cookie theft picture task, Vineland adaptive behavior scale (VABS)
Mynediad Ar-lein:http://www.sciencedirect.com/science/article/pii/S2214426922001136
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