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Efficient design and analysis of randomized controlled trials in rare neurological diseases: An example in Guillain-Barré syndrome

BACKGROUND: Randomized controlled trials (RCTs) pose specific challenges in rare and heterogeneous neurological diseases due to the small numbers of patients and heterogeneity in disease course. Two analytical approaches have been proposed to optimally handle these issues in RCTs: covariate adjustme...

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Detalles Bibliográficos
Autores principales: van Leeuwen, Nikki, Walgaard, Christa, van Doorn, Pieter A., Jacobs, Bart C., Steyerberg, Ewout W., Lingsma, Hester F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6382155/
https://www.ncbi.nlm.nih.gov/pubmed/30785890
http://dx.doi.org/10.1371/journal.pone.0211404