PROSPECTIVE HUNTINGTON AT RISK OBSERVATIONAL STUDY (PHAROS)

这个子项目是许多利用 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。 亨廷顿氏病是一种遗传性疾病，其特征是通常开始于成年期的运动、思维和判断的改变。 这项研究的参与者是那些被认为有患HD风险的人，因为他们的父母或兄弟姐妹患有这种疾病。 要符合条件，一个人必须没有任何患有这种疾病的迹象。 这是一项观察性研究。 这意味着本研究的研究参与者将接受定期检查，但您将不会接受研究药物或治疗。 研究中的所有参与者将完成相同的程序。 这些包括对健康，幸福，情绪和信仰的评估。 大多数评估是使用问卷进行的。 将采集血液样本以确定每个参与者是否携带HD基因，但这将完全保密。 研究人员和参与者都不会知道结果。 这项研究将为我们提供重要的信息，为未来的试验药物。 很少有研究在有遗传性疾病风险的个体（如HD患者的儿子，女儿或兄弟姐妹）中进行。 在我们能够在携带HD基因的健康个体中测试研究药物之前，我们必须解决一些涉及所有HD风险人群的重要问题，无论他们是否遗传了异常的HD基因。 我们希望在PHAROS中回答的一些问题包括： 1)在一群有患HD风险的人中，有多少人会在五到七年的时间里出现这种疾病的迹象？ 2)疾病的早期症状是什么，什么时候开始？ 3)研究人员用于检测HD发病的评估有多准确？ 4)什么因素会影响携带HD基因的人发病的年龄？'

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. Huntington's Disease is an inherited (genetic) disease characterized by changes in movement, thought and judgment that usually begin in adulthood. Participants in this study are people who are considered "at risk" for developing HD because they have had a parent or sibling with the disease. To be eligible, a person must not be showing any signs of having the disease. This is an observational study. This means that research participants in this study will be examined periodically, but you will receive no investigational drugs or treatments. All participants in the study will complete the same procedures. These include assessments of health, well-being, mood, and beliefs. Most assessments are done using questionnaires. A blood sample will be taken to determine whether each participant carries the gene for HD, but this will be kept entirely confidential. Neither the researchers nor the participants will know the results. This study will give us important information for future trials of investigational drugs. Little research has been done in individuals who are at risk for developing an inherited disease (like the son, daughter or sibling of a person with HD). Before we can test investigational drugs in healthy individuals who carry the HD gene, we have to address some important issues involving all persons at risk for HD, whether or not they have inherited the abnormal HD gene. Some questions we hope to answer in PHAROS include: 1) In a group of people who are at risk for HD, how many will develop signs of the illness over a five to seven year period? 2) What are the earliest signs of the illness, and when do they start? 3) How accurate are the evaluations that researchers use in detecting the onset of HD? 4) What factors influence the age at which a person carrying the HD gene develops the illness?'