The FDA has accepted uniQure's application for AMT-130, an experimental gene therapy for Huntington's disease, and set a May 2027 decision date. Here's what this milestone means, and what still needs to happen.

From sticky thoughts and pigs that model HD to DNA repair and promising drug trial results, October brought fresh insights into HD. Here's what you might have missed!

An experimental treatment for HD developed by Alnylam has shown long-lasting huntingtin lowering after just one dose, with encouraging early safety data. A pivotal trial is now planned for 2027 to figure out if this drug, brezosiran, can slow HD.