An experimental new gene therapy for Huntington’s disease has shown promising results in phase I/II trials, with 75 percent less disease progression observed in the patients receiving the treatment. If further trials continue to produce such positive results, we could be looking at a remarkable breakthrough for this incurable, life-limiting disease. The rest of this article is behind a paywall. Please sign in or subscribe to access the full content.

Key takeaways

AMT-130 is a new gene therapy that's just gone through phase I/II clinical trials in patients with Huntington's disease.

Huntington's is caused by a genetic mutation affecting the huntingtin protein, leading to progressive damage to the brain that is eventually fatal.

AMT-130 instructs brain cells to produce an RNA molecule th

See Full Page