2024
DOI: 10.1002/cpt.3466
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Longitudinal Analysis of Natural History Progression of Rare and Ultra‐Rare Cerebellar Ataxias Using Item Response Theory

Alzahra Hamdan,
Niels Hendrickx,
Andrew C. Hooker
et al.

Abstract: 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 for assessment and rating of ataxia (SARA). A longitudinal IRT model was built utilizing real‐world d… Show more

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