OPEN-LABEL FOLLOW-UP STUDY OF PHENYLBUTYRATE IN HD

这个子项目是众多研究子项目之一

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.