Clinical Need
Huntington's disease (HD) is a devastating, inherited, degenerative brain disorder that is progressive and always fatal and for which there is, at present, no effective treatment or cure. According to the National Institute of Neurological Disorders and Stroke, HD affects more than 30,000 people in the United States, with another 150,000 at risk of inheriting the deadly gene. The disease-causing gene produces a protein that is toxic to certain brain cells, and the subsequent neuronal damage leads to the movement disorders, psychiatric disturbances and cognitive decline that characterize this disease.
Our goal is to develop a curative solution designed to reduce or eliminate the accumulation of the Huntingtin protein to treat the as many as 250,000 people who carry the gene and are at risk. This would be a significant advance over current therapies that only affect signs and symptoms of the disease. |
