Huntington’s disease (HD) is a fatal brain disorder caused by a defect in the huntingtin (HTT) gene, which produces an extra-long protein. This defect causes the HTT protein to form harmful clumps, or ...

9 min read | January 2026 HD research recap: SKY-0515 shows promising safety data, community advocacy helps secure critical ...

Skyhawk Therapeutics shared results for SKY-0515, a drug designed to lower huntingtin. The drug appeared generally safe, with 60% reduction of huntingtin in blood at the highest dose tested. This data ...

5 min read |The Huntington’s disease community’s unprecedented mobilization helped secure a high-priority FDA meeting to discuss the path forward for AMT-130.

6 min read | Most people with HD report sleep problems, & new research suggests sleep quality is linked to many aspects of living with the disease. Wearables tracked people with HD for a year & ...

A new Huntington’s disease (HD) clinical trial has reached an important milestone: the first participants have now been dosed in POINT-HD, a Phase 1 study testing a new, selective huntingtin-lowering ...

Ever since large genetic studies in Huntington’s disease (HD) revealed that the longer the CAG expansion, the earlier symptoms appear, we’ve known that repeat length matters. Recent work has ...

SOM3355 is an investigational therapy aimed at managing multiple symptoms of Huntington’s disease (HD) and recently crossed two key regulatory milestones. In September, the European Medicines Agency ...

Scientists often use genetics, the study of DNA, to understand the cellular changes that cause disease. By comparing people’s DNA with their symptoms, they can pinpoint specific genetic differences, ...

A BLA, or Biologics Licensing Application, is the formal dossier of information that a company uses to request permission to market a biologic, which is a therapy created from living organisms, like ...