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 Hendrickx, Niels (Author)
, Mentré, France (Author)
, Hamdan, Alzahra (Author)
, Karlsson, Mats O. (Author)
, Hooker, Andrew C. (Author)
, Traschütz, Andreas (Author)
, Gagnon, Cynthia (Author)
, Schüle-Freyer, Rebecca (Author)
, Synofzik, Matthis (Author)
, Comets, Emmanuelle (Author)
, Chen, Xiaomei (Author)
, Heussen, Nicole Maria (Author)
, Hilgers, Ralf-Dieter (Author)
, Klockgether, Thomas (Author)
, Ryeznik, Yevgen (Author)
, Sverdlov, Oleksandr (Author)
,
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ARCA Study Group, EVIDENCE-RND consortium…”
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