On April 30, 2026, uniQure announced that it held a Pre-Submission Meeting with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) and plans to file a formal application ...

On May 5th, PTC Therapeutics released results from their ongoing Phase 2 PIVOT-HD clinical trial for PTC-518, now called votoplam. Excitingly, they announced that this trial met its primary endpoint – ...

10 min read | PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to ...

A new collaboration between researchers at Indiana University Southeast and Bellarmine University in Kentucky, both in the USA, aimed to investigate what happens when a physical therapist and a music ...

When caring for people affected by Huntington’s disease (HD), doctors often face major challenges due to the lack of precise tools to track how the disease progresses or to predict exactly when a ...

For individuals in the HD “grey zone” between 36-39 repeats, an understanding of when symptoms may arise and what kind of symptoms is critical. A new study sheds light on different sequence patterns ...

Skyhawk Therapeutics has announced results from a Phase 1 clinical study of its experimental huntingtin-lowering drug, SKY-0515. As with all Phase 1 studies, the main goal was to understand safety and ...

Huntington’s disease (HD) is caused by a repeated stretch of the genetic letters C-A-G within the huntingtin (HTT) gene above a critical number. If the repeats exceed 40, then signs and symptoms of HD ...

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 ...

On December 4, 2025, uniQure announced they have received the final meeting minutes from their October 29 pre-Biologics License Application (BLA) meeting with the FDA regarding AMT-130. The minutes ...

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 ...