Huntington’s disease has been successfully treated for the first time.
Huntington’s is a cruel illness: It manifests in patients’ 30s or 40s, when a misfolded protein in the brain starts killing neurons, causing symptoms that appear to be “a combination of dementia, Parkinson’s and motor neurone disease,” the BBC reported; it is always fatal. Huntington’s is caused by a single faulty gene, so a parent has a 50% chance of passing it to a child.
A new gene therapy, administered during brain surgery, slowed average progression by 75% in 29 subjects; since patients usually die within 20 years of diagnosis, the breakthrough could lead to decades of good-quality life. One researcher said the results were beyond “our wildest dreams.”