Article

Capturing clinical progression in multisystemic genetic ataxias: lessons from a prospective study of 884 patients with autosomal recessive or early-onset ataxia

2022-10-07

Abstract excerpt

<h4>Objective</h4> 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 relations to ataxia severity and patient-focused outcomes) across a large number of ataxias, and provide first natur...

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Literature Corpus work: 1f1a8bb9-71c1-54af-ad80-feefca1cd5e5
DOI: 10.1101/2022.10.05.22280687

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