On June 2, 2025, uniQure shared that they’re aligned with the FDA about moving forward with their HTT-lowering gene therapy, AMT‑130. If the next round of results from their ongoing trial continues to ...

The brain has plans in place when fires break out. Normally, a molecule called PKD1 rushes in to the rescue, but in ...

To date, the GeM-HD consortium has studied thousands of people with HD and hundreds of thousands of spelling changes in every ...

DNA-based drugs called antisense oligonucleotides, or ASOs, are now in multiple clinical trials in Huntington’s disease, aiming to lower production of the harmful mutant huntingtin protein in the ...

This week, we heard an update from Roche about their huntingtin-lowering therapy, tominersen, currently being tested in the GENERATION HD2 trial. An independent data monitoring committee (iDMC) that ...

There has been a lot of buzz in the Huntington’s disease (HD) space recently with multiple updates from companies testing many different drugs in the clinic. As these drugs move closer to seeking ...

Researchers have updated the system that classifies Huntington’s disease progression. Tracking progression in 4 stages will make clinical trial screening and data interpretation easier and faster, and ...

Good morning from the final day of the 2018 HD Therapeutics Conference! Two sessions today, the first focused on the protein made from the HD gene. The second includes updates on Huntingtin Lowering ...

A new study led by researchers from University College London has helped uncover some of the earliest changes that happen in people with the gene for Huntington’s disease (HD), long before obvious ...

A highly-anticipated scientific paper has landed! This new work challenges current theories in Huntington’s disease research, uncovering how runaway CAG repeats erode cell identity in certain types of ...

Imagine a tiny, microscopic surgeon moving through the body, making precise genetic repairs exactly where they’re needed. That’s the vision behind a groundbreaking new gene-editing delivery system ...