Huntington disease is a neurodegenerative disease that is caused by a buildup of the toxic huntingtin protein which causes the progressive breakdown of the nerve cells in the brain. Symptoms include psychiatric symptoms, impairments of voluntary movements, and dementia.
One in 10,000 Canadians suffer from the genetic condition, which has no known cure. The autosomal dominant disorder is caused by a single known genetic mutation, and a person needs only one copy of the defective gene to develop the disorder. Children of a parent with Huntington disease have a 50% chance of inheriting the condition.
International researchers, including a team from UBC, conducted a trial with 46 patients in Canada, the UK and Germany with early stage Huntington disease. These patients were either given medication or a placebo to see if the drug was effective in reducing the buildup of the toxic huntingtin protein.
The study showed that the R07234292 drug can safely decrease levels of the huntingtin protein in humans. Further trials are required before it can determine if the treatment is able to slow or halt the progression of the symptoms of Huntington disease.
Source: Science Daily
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