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Assessment of the Reliability, Responsiveness, and Meaningfulness of the Scale for the Assessment and Rating of Ataxia (SARA) for Lysosomal Storage Disorders

2024-07-19

Abstract excerpt

<h4>Objective</h4> To evaluate the reliability, responsiveness, and validity of the Scale for the Assessment and Rating of Ataxia (SARA) in patients with Lysosomal Storage Disorders (LSDs) who present with neurological symptoms and quantify the threshold for a clinically meaningful change. <h4>Methods</h4> We analyzed data from three clinical trial cohorts (IB1001-201, IB1001-202, IB1001-301) of patients with Niem...

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Literature Corpus work
137cc022-5e15-5e45-a9a1-2a4e6ff453f0
DOI
10.1101/2024.07.19.24310680
Open publication

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Assessment of the Reliability, Responsiveness, and Meaningfulness of the Scale for the Assessment and Rating of Ataxia (SARA) for Lysosomal Storage DisordersDOI 10.1101/2024.07.19.24310680
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