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ABSTRACT: Background
The Scale for the Assessment and Rating of Ataxia (SARA) is the reference clinical scale to assess the severity of cerebellar ataxia. In the context of upcoming therapeutic trials, a reliable clinical outcome is needed to assess the efficiency of treatments.Objective
The aim is to precisely assess and compare temporal dynamics of SARA and a new f-SARA.Methods
We analyzed data from four cohorts (EUROSCA, RISCA, CRC-SCA, and SPATAX) comprising 1210 participants and 4092 visits. The linearity of the progression and the variability were assessed using an ordinal Bayesian mixed-effect model (Leaspy). We performed sample size calculations for therapeutic trials with different scenarios to improve the responsiveness of the scale.Results
Seven of the eigh
SUBMITTER: Moulaire P
PROVIDER: S-EPMC9851985 | biostudies-literature | 2023 Jan
REPOSITORIES: biostudies-literature