Huntington’s disease treated for first time using gene therapy
Elie Dolgin • September 25, 2025
Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.
A one-time gene therapy can markedly slow the progression of Huntington’s disease, potentially paving the way for the first ever treatment to alter the course of this rare, inherited brain disorder.
Continue reading at Nature.
Jaw-dropping patient videos and miraculous testimonials ignited a frenzy in China over a technique that aims to improve drainage from the brain. Now it is entering trials worldwide.
But politics could derail the one thing to unlock it.