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Item performance of the scale for the assessment and rating of ataxia in rare and ultra‐rare genetic ataxias by Alzahra Hamdan, Andrew C. Hooker, Xiaomei Chen, Andreas Traschütz, Rebecca Schüle, ARCA Study Group, EVIDENCE‐RND consortium, Matthis Synofzik, Mats O. Karlsson
Published 2024-08-01Get full text
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Reflection on clinical and methodological issues in rare disease clinical trials. by Johan Verbeeck, Martin Geroldinger, Joakim Nyberg, Konstantin E. Thiel, Andrew C. Hooker, Arne C. Bathke, Johann W. Bauer, Geert Molenberghs, Martin Laimer, Georg Zimmermann
Published 2025-06-01Get full text
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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 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
Published 2025-07-01Get full text
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