From stopping CAG repeats from growing to finding new ways to tackle harmful huntingtin, scientists at the EHDN Clinical Research Congress 2026 shared exciting ideas for the next generation of HD treatments. Here's what's coming down the pipeline.

Skyhawk's FALCON-HD trial has dosed the first participants in the US with the drug, SKY-0515. The once-daily pill targets two drivers of HD, but does it actually slow disease? That's the big question this study hopes to answer.

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.