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。 本项目的目的是验证弗里德赖希共济失调（FRDA）患者的临床结局指标。 迄今为止，缺乏可靠和敏感的测量方法，这是开始治疗试验的限制因素。该研究将使用多种临床评估工具，包括新设计的FRDA评级量表（法尔斯），9孔钉测试和定时25英尺步行，跟踪约200名处于不同疾病阶段的个体。 还将进行对比度字母敏锐度测量和言语定量测量。 所有的措施都是非侵入性的，许多是在详细的神经系统检查期间进行的评估。还将管理健康相关的生活质量，以确定患者的状况如何影响其生活质量以及这种情况如何随时间变化。 将在三年内每年对参与者进行跟踪。 他们将被允许参加所有这些研究，或者如果他们选择只参加一部分。

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.