• The upcoming clinical trial wouldn’t be possible without a new ultra-sensitive test that allows scientists to measure the mutant protein in the cerebrospinal fluid of Huntington’s disease patients.
  • Researchers found that the level of mutant protein is higher in patients with more advanced disease. The concentration of mutant protein also predicts the severity of movement and cognitive problem in patients, according to the study.
  • The drug, which was developed by California-based Isis Pharmaceuticals, will be administered into the patients’ cerebrospinal fluid to “turn off” the mutant gene in the brain.

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