OPEN-LABEL FOLLOW-UP STUDY OF PHENYLBUTYRATE IN HD

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。 亨廷顿病是一种常染色体显性遗传的、一致进行性神经退行性疾病，临床上以运动障碍（舞蹈病）、人格改变和痴呆为特征。 在美国大约有30，000人有HD的临床表现，另外150，000名健康人被认为有立即发展HD的风险。 本临床试验的目的是评价苯丁酸盐每日15克在亨廷顿病（HD）受试者中的安全性、耐受性和短期临床影响。 此外，这项研究将作为后续试验的基础，旨在专门解决苯丁酸盐减缓或停止HD进展的能力。

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 autosomal dominant, uniformly progressive neurodegenerative disorder characterized clinically by movement disorder (chorea), personality changes, and dementia. Approximately 30,000 people in the United States have clinical manifestations of HD, and an additional 150,000 healthy people are thought to be immediately at risk to develop HD. The purpose of this clinical trial is to evaluate the safety, tolerability and short-term clinical impact of 15-grams daily phenylbutyrate in subjects with Huntington's disease (HD). In addition, this study will serve as a basis for subsequent trials designed to specifically address phenylbutyrate's ability to slow or halt the progression of HD.