CLINICAL OUTCOME MEASURES IN FRIEDREICH'S ATAXIA

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 本研究的目的是验证Friedreich共济失调（FA）患者的临床结局指标。 缺乏可靠和敏感的措施是限制因素，在开始治疗试验的FA。 这项研究的目的是：1）FA中的神经功能缺损可通过定量性能指标进行测量; 2）健康相关生活质量指标将与目标1中设计的疾病活动性临床指标显著相关; 3）神经功能缺损的简单性能指标将对FA患者随时间的变化做出反应。

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.