AANS Neurosurgeon | Volume 27, Number 2, 2018


Potential Treament for Huntington's Disease, Found Effective, Safe in Mice, Monkeys, Enters Clinical Testing

Huntington’s disease is a rare, hereditary disease. This disease causes uncontrollable movements, loss of intellectual abilities, emotional problems and eventually death. As it is hereditary, this disease is passed on from generation to generation through a mutation in the huntingtin gene. Due to the mutation, the production of the disease-causing huntingtin protein occurs. Furthermore, anyone who inherits the gene will eventually develop the disease. A drug that would be the first of its kind to target the cause of Huntington’s disease has been deemed effective and safe when tested in mice and monkeys. “It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease,” said clinical study principal investigator Blair R. Leavitt, MD, of the University of British Columbia in Vancouver. “Right now we only have treatments that work on the symptoms of the disease.” Click here to read more. 

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