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Comparing Randomized Trial Designs to Estimate Treatment Effect in Rare Diseases with Longitudinal Models: a Simulation Study Showcased by Autosomal Recessive Cerebellar Ataxias Using the SARA Score

Niels Hendrickx,France Mentré, Alzahra Hamdan,Mats O Karlsson,Andrew C Hooker,Andreas Traschütz, Cynthia Gagnon,Rebecca Schüle, ARCA Study Group, EVIDENCE-RND Consortium,Matthis Synofzik, Emmanuelle Comets

medrxiv（2025）

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