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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Detalles Bibliográficos
Autores principales: Chapman, Kimberly A., MacEachern, Devon, Cox, Gerald F., Waller, Mavis, Fogarty, Jeanine, Granger, Suzanne, Stepanians, Miganush, Waisbren, Susan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9842695/
https://www.ncbi.nlm.nih.gov/pubmed/36659999
http://dx.doi.org/10.1016/j.ymgmr.2022.100953

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9842695/
https://www.ncbi.nlm.nih.gov/pubmed/36659999
http://dx.doi.org/10.1016/j.ymgmr.2022.100953

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