CLINICAL OUTCOME MEASURES IN FRIEDREICH'S ATAXIA

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 该项目的目的是验证Friedreich共济失调(FRDA)患者的临床结果。到目前为止，缺乏可靠和灵敏的测量方法，这是启动治疗试验的限制因素。这项研究将使用多种临床评估工具对大约200名处于不同疾病阶段的人进行跟踪调查，这些工具包括新设计的FRDA评价表(FARS)、9洞钉住测试和定时25英尺步行。还将进行对比字母敏锐度的测量和语音的量化测量。所有的测量都是非侵入性的，许多都是在详细的神经学检查中进行的评估。与健康相关的生活质量也将被实施，以确定患者的状况如何影响他们的生活质量，以及这种影响如何随着时间的推移而变化。参与者将在三年内每年接受跟踪调查。他们将被允许参与所有这些研究，或者如果他们愿意，只允许参与部分研究。

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 project is to validate clinical outcome measures in individuals with Friedreich's Ataxia (FRDA). To date, there is a lack of reliable and sensitive mesures which is the limiting factor in the initiation of therapeutic trials. The study will follow approximately 200 individuals at various stages of illness using multiple clinical assessment tools including a newly devised FRDA rating scale (FARS), the 9-hole peg test, and a timed 25-foot walk. A measure of contrast letter acuity and quantative measures of speech will also be performed. All the measures are non-invasive and many are assessments performed during a detailed neurological examination. A health related quality of life will also be administered to determine how patients' conditions impact their quality of life and how this changes over time. Participants will be followed annually over a period of three years. They will be allowed to participate in all of these studies or only a portion if they choose.