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 ...

Long repetitive sequences of C-A-G letters in the DNA code are associated with at least 12 genetic diseases, including Huntington’s disease (HD). A group of scientists in Massachusetts, USA, have ...

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 ...

Greetings from Day 2 of the CHDI HD Therapeutics Conference! HDBuzz continues to provide summaries of the exciting talks from HD scientists gathered in Palm Springs from around the world. This day’s ...

We all know heart health matters – but what if the same habits that keep your heart strong could also protect your brain? A new study reveals a link between cardiovascular health and lower levels of ...

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 ...

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 ...

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 ...