How is disease progress in Friedreich's ataxia best measured? A study of four rating scales

Abstract

BACKGROUND: Friedreich's ataxia (FRDA), the most common genetic cause of ataxia, is characterised by progressive neurodegeneration and cardiomyopathy. Initial treatments are likely to slow progression rather than reverse morbidity. An appropriate and sensitive scale to measure disease progress is critical to detect the benefit of treatments. OBJECTIVE: To compare the Friedreich Ataxia Rating Scale (FARS) with other scales proposed as outcome measures for FRDA. METHODS: 76 participants were assessed with the FARS and the International Cooperative Ataxia Rating Scale (ICARS) and 72 of these participants were also assessed with the Functional Independence Measure and the Modified Barthel Index...