As 2025 winds down, HD research is more active, more interdisciplinary, and more globally connected than ever. Whether it’s genetic modifiers, new drug targets, or next-generation clinical trials, ...

Usually, there are helper proteins, called “chaperones,” that work throughout the cell to make sure other proteins fold correctly and prevent them from clumping. However, in diseases like HD, this ...

SOM3355 is still an investigational drug, and only a Phase 3 trial can determine whether it is safe and effective enough to seek approval. But the combined momentum from the EMA and FDA reflects a ...

Last month, we relayed positive news from uniQure’s trial testing AMT-130, a gene therapy delivered via brain surgery to lower huntingtin (HTT). Data released by uniQure in June suggested AMT-130 was ...

For those who were following the live tweets from HDBuzz about the CHDI HD Therapeutics Conference or tuned in to the HDSA Convention, we may have caught your attention with the new HD staging system.

UniQure has announced positive top-line results from its Phase I/II trial of AMT-130, a one-time gene therapy being tested in people with Huntington’s disease (HD). Topline data is a summary of the ...

Welcome to Day 2 of the Hereditary Disease Foundation (HDF) conference! The morning was spent listening to an interview between a neurologist and their patient living with HD. All HDF meetings begin ...

The past 4 weeks have been a whirlwind in the Huntington’s disease (HD) community. On September 25th we had an update from uniQure about a drug they’re testing for HD in ongoing clinical trials. The ...

Imagine the gene that causes Huntington’s disease (HD) as a vast river. At its source is the CAG repeat – a genetic letter code that dictates how the river will flow. As the river moves downstream, it ...

A recent clinical trial successfully tested the safety of CRISPR gene editing to reduce the amount of a toxic protein in patients with Familial Transthyretic (TTR) Amyloidosis. Although this study is ...

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 ...