Ricardo Dolmetsch, PhD, President of Research and Development at uniQure, gives an overview of Huntington disease.
As Dr. Dolmetsch explains, Huntington disease is an inherited, neurodegenerative disorder that usually begins to be symptomatic starting at around 40 years of age. Persons with Huntington disease suffer from a progressive movement disorder, cognitive decline, psychiatric disturbances, and behavioral symptoms. All symptoms progress which eventually leads to immobility, dementia, and premature death.
Last year, two of the leading clinical trials involving possible treatments for Huntington disease were terminated. Fortunately, uniQure recently announced positive safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase 1/2 clinical trial of AMT-130, the first-ever AAV gene therapy for Huntington’s disease.
To learn more about Huntington disease and other rare neurological disorders, visit checkrare.com/diseases/neurology