Dr. Blair Leavitt, a professor in UBC’s department of medical genetics heading up the city’s part of the international clinical trial, said the drug has the potential to directly treat the underlying cause of Huntington’s disease.
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, said Leavitt.
http://metronews.ca/news/vancouver/1333888/new-drug-for-huntingtons-disease-to-be-tested-in-vancouver/