Comparing randomized trial designs to estimate treatment effect in rare diseases with longitudinal models: a simulation study showcased by Autosomal Recessive Cerebellar Ataxias us... by Niels Hendrickx, France Mentré, Alzahra Hamdan, Mats O. Karlsson, Andrew C. Hooker, Andreas Traschütz, Cynthia Gagnon, Rebecca Schüle, Matthis Synofzik, Emmanuelle Comets, ARCA Study Group, EVIDENCE-RND consortium

Get full text