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

<h4>Background</h4>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:...

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Bibliographic Details
Main Authors: Nikki van Leeuwen, Christa Walgaard, Pieter A van Doorn, Bart C Jacobs, Ewout W Steyerberg, Hester F Lingsma
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2019-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0211404