We at uniQure are fully committed to the clinical development of AMT-130, the first AAV gene therapy to be investigated in Huntington’s disease. We are deeply appreciative of the support that we’ve received from the community and for the pioneering individuals who have volunteered to be a part of our clinical studies. Only through such collaboration can we strive to develop safe and effective treatments to modify the course of Huntington’s disease.
Huntington’s disease (HD) is a rare, fatal, neurodegenerative genetic condition that affects motor function and leads to behavioral symptoms and cognitive decline in adults, resulting in total physical and mental deterioration over a 12 to 15-year period. Huntington’s disease affects approximately 75,000 people in the U.S., Europe and UK.
Huntington’s disease (HD) is an inherited condition that causes the progressive breakdown of brain cells. Buildup of mutant huntingtin protein is thought to cause the disease. The striatum is a core structure of the brain that is first affected in people with Huntington’s. This structure is critical for motor function and reward- and goal-oriented behavior. Loss of brain cells in the striatum leads to the following problems:
- Motor function issues
- Cognitive dysfunction
- Psychiatric disturbances
Despite the discovery of the gene that causes HD, there are no therapies available to treat the disease, delay onset, or slow progression of a patient’s decline.