CLINICAL OUTCOME MEASURES IN FRIEDREICH'S ATAXIA

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The purpose of this study is to validate clinical outcome measures in individuals with Friedreich Ataxia (FA). The lack of reliable and sensitive measures is the limiting factor in initiation of therapeutic trials in FA. The aims of the study are: 1) neurologic impairment in FA can be measured through quantitative performance measures; 2) health-related quality of life measures will correlate significantly with clinical measures of disease activity designed in Aim 1; 3) simple performance measures of neurologic impairments will be responsive to change over time in patients with FA.