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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Principais autores:	Kimberly A. Chapman, Devon MacEachern, Gerald F. Cox, Mavis Waller, Jeanine Fogarty, Suzanne Granger, Miganush Stepanians, Susan Waisbren
Format:	Artigo
Jezik:	Inglês
Izdano:	Elsevier 2023-03-01
Serija:	Molecular Genetics and Metabolism Reports
Teme:	Methylmalonic acidemia Propionic acidemia Neuropsychological testing Cookie theft picture task, Vineland adaptive behavior scale (VABS)
Online dostop:	http://www.sciencedirect.com/science/article/pii/S2214426922001136
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