OPEN-LABEL FOLLOW-UP STUDY OF PHENYLBUTYRATE IN HD

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。 亨廷顿病是一种常染色体显性、均匀进行性神经退行性疾病，临床特征为运动障碍(舞蹈症)、人格改变和痴呆症。在美国，大约有30,000人有HD的临床表现，另有150,000名健康的人被认为立即有患HD的风险。这项临床试验的目的是评估每天服用15克苯丁酸盐对亨廷顿病(HD)患者的安全性、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，专门研究苯丁酸酯延缓或阻止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.