Article
Longitudinal Analysis of Natural History Progression of Rare and Ultra-Rare Cerebellar Ataxias Using Item Response Theory.
Clinical pharmacology and therapeutics - 1 Dec 2024
Hamdan Alzahra, Hendrickx Niels, Hooker Andrew C, Chen Xiaomei, Comets Emmanuelle, Traschütz Andreas, Schüle Rebecca, Mentré France, Synofzik Matthis, Karlsson Mats O
Abstract excerpt
Degenerative cerebellar ataxias comprise a heterogeneous group of rare and ultra-rare genetic diseases. While disease-modifying treatments are now on the horizon for many ataxias, robust trial designs and analysis methods are lacking. To better inform trial designs, we applied item response theory (IRT) modeling to evaluate the natural history progression of several ataxias, assessed with the widely used scale...
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