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Huntington's Disease




What is the most promising new breakthrough or research on Huntington`s Disease.


There is much exciting Huntington's disease research going on right now. There are several clinical trials underway at several centers around the country looking at agents that may slow the course of disease progression and some looking at new ways to treat disease symptoms. There is still no cure for the disorder. I think one of the more promising approaches involves discovering ways to halt production of the abnormal Huntington disease protein by the cells in the brain. One approach to this currently being studied in animal models (and is being considered for trials in humans in the near future), involves the use of a type of blocking or interfering molecule sometimes referred to a siRNA. 

There are several other exciting new treatments on the horizon. As Huntington's disease is a relatively rare disorder, finding a cure for this disease involves the participation of basic scientists, HD clinical researchers and the participation of patients and their families.

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Response by:

Sandra  K Kostyk, MD, PhD Sandra K Kostyk, MD, PhD
Assistant Professor of Neuroscience
Clinical Assistant Professor of Neurology
College of Medicine
The Ohio State University