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Biomarker gene target identified to track Huntington's disease

October 4, 2011 - Washington

Scientists have identified a biomarker gene that could be used to follow the activity of Huntington's disease as well as the response patients might have to neuroprotective treatments.

Huntington's disease, a devastating genetic disorder that causes degeneration of nerve cells in the brain, affects more than 15,000 Americans, and at least 150,000 are at risk of developing the disease.

There is no known cure or treatment for the disease-which starts with changes in mood, judgment, memory, and other cognitive functions and inevitably leads to increasing physical disability, Dementia and death.

The new research describes the discovery and validation of a Blood test that could assist in the monitoring of disease activity and in the evaluation of new medications.

"We know how to diagnose HD. What we don't have, however, is a simple test to tell us whether the disease is active and progressing or responding to new medications," said Clemens Scherzer, MD, in the Center for Neurologic Diseases at Brigham and Women's Hospital.

"Such a test would be critical for making clinical trials more efficient. We are excited about the potential of our discovery.

"The next challenge will be to develop this prototype biomarker into a test that is useful in drug trials," he added.

The study has been published in the Proceedings of the National Academy of Sciences.


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