Responsiveness of the Scale for the Assessment and Rating of Ataxia and Natural History in 884 Recessive and Early Onset Ataxia Patients

Objective The Scale for the Assessment and Rating of Ataxia (SARA) is the most widely applied clinical outcome assessment (COA) for genetic ataxias, but presents metrological and regulatory challenges. To facilitate trial planning, we characterize its responsiveness (including subitem‐level relation...

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Veröffentlicht in:	Annals of neurology 2023-09, Vol.94 (3), p.470-485
Hauptverfasser:	Traschütz, Andreas, Adarmes‐Gómez, Astrid D., Anheim, Mathieu, Baets, Jonathan, Brais, Bernard, Gagnon, Cynthia, Gburek‐Augustat, Janina, Doss, Sarah, Hanağası, Haşmet A., Kamm, Christoph, Klivenyi, Peter, Klockgether, Thomas, Klopstock, Thomas, Minnerop, Martina, Münchau, Alexander, Renaud, Mathilde, Santorelli, Filippo M., Schöls, Ludger, Thieme, Andreas, Vielhaber, Stefan, Warrenburg, Bart P., Zanni, Ginevra, Hilgers, Ralf‐Dieter, Synofzik, Matthis
Format:	Artikel
Sprache:	eng
Schlagworte:	Genetics Life Sciences
Online-Zugang:	Volltext
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