September 2026: This month in Huntington’s disease research

September was another busy month for Huntington’s disease (HD) research. Scientists explored new ways to track changes in the brain, took a huge-data approach to understanding the DNA repeats at the heart of HD, and showcased technologies that are transforming what researchers can measure in the laboratory. Meanwhile, new clinical trialclinical trial Very carefully planned experiments designed to answer specific questions about how a drug affects human beings data provided an encouraging update on an experimental huntingtin-lowering drug. This month also brought important research into nutrition and mental health, reminding us that improving life with HD means understanding and supporting the whole person. Let’s get into this month’s updates!

Seeing and hearing HD differently

A major challenge for HD researchers is finding sensitive ways to measure how the disease changes over time. Better measurements could help doctors understand an individual’s disease more precisely and, importantly, make clinical trials faster and more informative. 

One September study looked at visual thinking skills, our ability to process and make sense of what we see. Researchers found that changes in these abilities may contain useful information about future HD progression. Before a clinical diagnosis, poorer performance on some visual thinking tests was associated with greater changes later on for movement symptoms. In people with early HD, visual attention and spatial abilities were instead linked with later changes in cognition. These tests aren’t ready to replace established HD assessments just yet, but they could eventually add another tool to the toolbox for tracking disease.

Researchers are also exploring whether they can listen to the electrical activity of the brain. A review of 23 studies examined electroencephalography, better known as EEG, which measures brain waves using electrodes placed on the scalp. Across studies, people with HD showed changes in patterns of brain activity and some of these differences were associated with established measures of HD progression. EEG is non-invasive and already widely used in medicine. There’s more work to do to standardise how it is used in HD, but these findings suggest brain waves could one day contribute to a suite of biomarkers helping researchers track HD more sensitively.

Bigger datasets and better tools

September also gave us a glimpse at the increasingly sophisticated toolkit being used to tackle HD. Five scientists shared some of the technologies powering their research, highlighting how advances in experimental methods are allowing researchers to ask questions that would have been difficult or impossible just a few years ago. This matters because there probably won’t be one experiment, technology, or clever trick that cracks HD. Progress often comes from combining different approaches to understand the disease from multiple angles. Better tools mean researchers can see biology in greater detail, test ideas more rigorously, and hopefully move promising discoveries towards treatments more efficiently.

We also learned about a study analysing DNA sequencing data from more than 900,000 people that examined how repetitive stretches of DNA change over time. The CAG repeatCAG repeat The stretch of DNA at the beginning of the HD gene, which contains the sequence CAG repeated many times, and is abnormally long in people who will develop HD that causes HD can expand in some cells as we age, a process called somatic expansionsomatic expansion A process in which the CAG repeat in the Huntingtin gene can change over a person’s lifetime in some cells of the body, particularly in the brain., which is increasingly thought to be important in determining when HD symptoms emerge. The enormous new study showed that age-related repeat expansion isn’t unique to the huntingtin gene. Repeat sequences elsewhere in our DNA can also be unstable, with their behaviour influenced by repeat length, sequence and DNA repair machinery. HD research has helped drive our understanding of this biology, and these huge datasets now provide researchers with new opportunities to uncover the fundamental rules governing our DNA.

Encouraging signs for huntingtin lowering

There was also news from the clinic. Skyhawk Therapeutics reported final 15-month Phase 1/2 data for SKY-0515, an oral drug designed to alter RNARNA the chemical, similar to DNA, that makes up the ‘message’ molecules that cells use as working copies of genes, when manufacturing proteins. processing and lower huntingtin. The update suggests the drug continues to do what it was designed to do, lowering huntingtin as well as PMS1, a protein involved in the DNA repair machinery linked to somaticsomatic relating to the body CAG expansion. Skyhawk also reported encouraging signals on clinical measures, although this small early-stage study was not designed to establish whether SKY-0515 slows HD. That question now moves to the much larger FALCON-HD Phase 2/3 study, which is already underway. 

Looking beyond the brain

Two September studies broadened the picture further. Research on nutrition found that signs of nutritional difficulties were more common in people with HD than in people without HD, although estimates varied depending on how nutrition was measured. Poorer nutritional scores were also associated with more advanced disease, reduced daily functioning, and poorer cognitive performance. The findings don’t mean nutrition causes HD progression, but they reinforce something immediately relevant to HD care: monitoring weight, swallowing, food intake, and nutritional needs can be an important part of supporting health and quality of life.

Finally, research using Enroll-HD data highlighted a serious disparity in mental health. Indigenous North American participants had more than twice the odds of reporting a history of suicidal thoughts compared with White non-Hispanic participants. The study was relatively small, but it draws attention to communities historically underrepresented in HD research and to the social, historical, and healthcare factors that shape health alongside genetics. Importantly, identifying disparities creates opportunities to address them through more inclusive research, culturally grounded care, trusted community partnerships, and better access to mental health support.

Progress from many directions

Taken together, September’s stories show just how broad HD research has become. Scientists are finding better ways to measure the disease, building increasingly powerful experimental tools, learning fundamental lessons from enormous genetic datasets, and testing new treatments in people. At the same time, research is expanding its focus beyond molecules and movement symptoms to nutrition, mental health, and disparities in care. 

Summary: 

• New ways to track HD: Visual thinking tests and EEG brain-wave measurements could add sensitive, non-invasive tools for monitoring disease progression. 
• Learning from DNA at enormous scale: Analysis of more than 900,000 genomes is revealing the rules governing age-related repeat expansion, with important implications for HD.
• Treatment research keeps moving: 15-month SKY-0515 data show continued huntingtin lowering and encouraging clinical signals, while the larger FALCON-HD study will test whether these translate into benefit.
• Supporting the whole person: New studies highlight nutrition, mental health, and equitable access to care as important pieces of the HD puzzle.