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FDA approves first drug for Huntington's disease
Date:8/15/2008

ppear to help other symptoms of Huntington's disease, and it does not slow the progression of the disease or stop the underlying disease process. But most neurologists feel that overall, the benefits of the drug outweigh the side effects, especially considering that there has been no medication approved for the treatment of Huntington's patients.

Huntington's disease usually strikes people in their 30s and 40s, though some patients are affected as early as childhood, while others aren't affected until their older years. The disease is caused by the death of brain cells known as medium spiny neurons, which are killed off by a mutant protein. The disease brings with it an array of difficulties besides chorea, including cognitive problems, changes in personality, and psychiatric problems like depression. As many as one-quarter of patients with the disease attempt suicide, and many suffer from progressive cognitive decline. Unlike Alzheimer's disease, where patients usually lose their memory and insight into their disease at some point, most Huntington's patients understand exactly what is happening to them throughout most of their illness.

Virtually everyone with the disease had a parent with the disease, and children of a person with Huntington's have a 50-percent chance of inheriting the disease. Fifteen years ago the gene that causes the disease was identified by Wexler and colleagues, and now a simple blood test can tell people whether they will develop the disease or not. But since there is no way known to prevent the disease or slow its progression, and for other reasons as well, many patients decline the test, instead waiting to see if they develop symptoms like the ones they witnessed in a parent.

Prestwick Pharmaceuticals of Washington, D.C. owns the rights to develop and sell tetrabenazine in the United States. The medication was originally developed in the 1950s to treat psychosis, but was quickly pushed aside by more effective me
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Contact: Tom Rickey
tom_rickey@urmc.rochester.edu
585-275-7954
University of Rochester Medical Center
Source:Eurekalert

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