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HDBuzz @hdbuzzfeed.bsky.social · 30/09/2026

uniQure reports that after 4 years, people given high-dose AMT-130 continue to show signs of slower HD progression, providing hope for their recent regulatory submission. But the new analyses are nuanced. We unpack the hopeful but complex results in this article → en.hdbuzz.net/amt-130-at-f...

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HDBuzz @hdbuzzfeed.bsky.social · 28/09/2026

September brought progress across the Huntington’s disease research landscape. From brain waves and visual thinking to DNA repeats, nutrition, inclusive care, new technologies, and encouraging SKY-0515 data, we round up a busy month in HD research. Full article here → en.hdbuzz.net/september-20...

Picture of a toolbox with title, “September 2026: This month in Huntington’s disease research

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HDBuzz @hdbuzzfeed.bsky.social · 24/09/2026

The CAG repeat that causes Huntington’s disease (HD) has a tendency to grow with age. New research on over 900,000 people uncovers parallel patterns and finds CAG growth can happen in many other parts of the genome. #hdresearch Read the full article → en.hdbuzz.net/studying-stu...

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HDBuzz @hdbuzzfeed.bsky.social · 21/09/2026

Unique research highlights an overlooked mental health disparity among Indigenous North American people with Huntington’s disease (HD) - and the need for care grounded in trust, community partnership, and cultural understanding. Read more here: en.hdbuzz.net/highlighting...

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HDBuzz @hdbuzzfeed.bsky.social · 16/09/2026

Skyhawk Therapeutics shared data for SKY-0515, an experimental once-daily pill that targets both huntingtin and somatic instability, making it a possible “two-for-one” drug to treat HD. Read more about this exciting news on HDBuzz → en.hdbuzz.net/encouraging-...

Graphic of a megaphone with the words, "Encouraging news from Skyhawk Therapeutics!" above.

Picture of pills from a pill bottle with words on text boxes: "A potential 2-for-1 approach to treat HD is safe and shows potential! SKY-0515 is an experimental once-daily pill that aims to lower huntingtin AND target somatic instability."

Close up photo of a doctor checking lab results. Words in text boxes read, "In the Phase 1/2 study, SKY0515 did just that, and showed positive changes in tests of movement & thinking." But will that add up to a treatment that slows HD?"

Text box: "Skyhawk's Phase 2/3 study, FALCON-HD, will tell us whether the Phase 1/2 study's promising signs hold up in a larger, placebo-controlled trial."

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HDBuzz @hdbuzzfeed.bsky.social · 14/09/2026

What if nutrition is one of the warning lights we should be checking earlier in Huntington’s disease? New research suggests simple nutrition measures may help identify when people with HD need extra support. en.hdbuzz.net/food-for-tho...

Photo of puzzle pieces on a dinner plate. Title reads, "Food for Thought: The Importance of Nutrition in Huntington's Disease".

Graphic of a wind cloud blowing at a woman holding onto an umbrella. Text reads, "Living with HD can be like walking into a strong headwind..."

Text continues, "... you're still moving forward, but it takes more energy. So a new study on nutrition in people with HD set out to discover if..."

Text continues, "1. malnutrition is more common in HD,
2. malnutrition is linked with symptoms such as movement and thinking changes, and
3. simple nutrition checks might help care teams spot when extra support is needed."

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HDBuzz @hdbuzzfeed.bsky.social · 10/09/2026

A review of 23 studies suggests that EEG could offer a non-invasive way to track brain changes across the course of Huntington’s disease (HD). #hdresearch Read all about it → en.hdbuzz.net/could-brain-...

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HDBuzz @hdbuzzfeed.bsky.social · 07/09/2026

Five Huntington’s disease (HD) researchers discuss the cutting-edge techniques driving their work, accelerating discovery and guiding development of treatments. #hdresearch Read all about it → en.hdbuzz.net/hot-techniqu...

Five research scientists compare notes. Title reads "Hot Techniques in HD Research: Five HD researches discuss the cutting-edge techniques driving their work."

Scientist looks in a microscope. "From reading DNA molecule by molecule..."

"... to visualizing proteins and neurons in remarkable detail,"

Man with a camera over the words, "these technologies are giving scientists an increasingly complete picture of how HD begins and progresses."

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HDBuzz @hdbuzzfeed.bsky.social · 03/09/2026

What if Huntington’s disease (HD) changes how we “see” the world before we even notice symptoms? New research suggests visual thinking skills may help track HD progression. en.hdbuzz.net/keeping-an-e...

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HDBuzz @hdbuzzfeed.bsky.social · 02/09/2026

NEWS! uniQure has submitted AMT-130 for regulatory approval in the US and UK. This drug is NOT yet approved; regulators must now review the evidence. Read more about this milestone here -> uniqure.gcs-web.com/node/12991/pdf and on HDBuzz here -> en.hdbuzz.net/the-other-sh...

Graphics of a megaphone and arrow pointing to words: "Press Release: uniQure has submitted AMT-130 for regulatory approval in the US & UK"

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HDBuzz @hdbuzzfeed.bsky.social · 31/08/2026

August was a busy month for Huntington’s disease (HD) research! Setbacks, new trials, a conference, and the HDBuzz Prize for Young Science Writers! Catch up on everything in our monthly roundup: en.hdbuzz.net/august-2026-...

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HDBuzz @hdbuzzfeed.bsky.social · 27/08/2026

A new Huntington's disease (HD) clinical trial has officially begun! It will test whether the drug pridopidine can slow the progression of HD. For more details, read our article: en.hdbuzz.net/first-partic...

Over a photo of medication pills is the text: "Analysis of a previous trial’s data suggested that people who were not taking antidopaminergic medications (ADMs) appeared to have greater benefit from pridopidine than those who were."

Over a photo of medication pills is the text "PRECISE-HD is a new confirmatory trial specifically designed to test that hypothesis."

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Reposted by HDBuzz

Huntington's Disease Foundation @hdfcures.bsky.social · 25/08/2026

Rachel Harding & Leora Fox from @hdbuzzfeed.bsky.social will join us on 9/9 to share the buzz from HDF’s recent HD2026 Symposium. They'll recap some of the exciting science from the meeting and hold a live Q&A. Register Now: us06web.zoom.us/.../501.../W... #curehd #HD2026 #researchspotlightwebinars

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Reposted by HDBuzz

Andy Gates @andygates.bsky.social · 20/08/2026

If you've got stage 2 HD and want to do a science, here's a new Phase 1 study for a potential injectable therapy. They've got a cunning plan for getting treatments across the blood-brain barrier.

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HDBuzz @hdbuzzfeed.bsky.social · 24/08/2026

New research points to HTT1a, a toxic shortened form of huntingtin (HTT), as a key driver of disease and a promising therapeutic target in Huntington’s disease (HD). Read the full article here: en.hdbuzz.net/taking-a-sho...

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HDBuzz @hdbuzzfeed.bsky.social · 20/08/2026

New Huntington’s disease (HD) trial begins! A Phase 1 study is testing SRP-1005, a huntingtin-lowering therapy given by injection under the skin. It’s early days, but this approach could add an important new arrow to the HD drug development quiver. More details here -> en.hdbuzz.net/a-shot-in-th...

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Reposted by HDBuzz

Jose Lucas Lab @jjlucaslab.bsky.social · 20/08/2026

Thanks, Leora Fox & Rachel Harding @hdbuzzfeed.bsky.social, for the excellent summary of my presentation at @hdfcures.bsky.social Meeting on the #HUNTIAM clinical trial (Thiamine+Biotin) for HD, including the evidence that originated it. 👇👇👇 en.hdbuzz.net/hd2026-milto... @cbm-csic-uam.bsky.social

en.hdbuzz.net

Thank you – HDBuzz

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HDBuzz @hdbuzzfeed.bsky.social · 17/08/2026

Announcing the 2026 HDBuzz Prize for Young Science Writers – sponsored by the Hereditary Disease Foundation! Submission details here: en.hdbuzz.net/the-2026-hdb...

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HDBuzz @hdbuzzfeed.bsky.social · 14/08/2026

Day 3 of #HD2026 put the huntingtin protein (HTT) under the microscope. Researchers shared new insights into HTT1a, next-generation HTT-lowering approaches, somatic expansion, brain circuitry, and why boosting healthy HTT might help. Read our roundup here: en.hdbuzz.net/hd2026-milto...

The final day of HD2026 put the huntingtin protein (HTT) under the microscope.

Researchers shared:

- new insights into HTT1a
- next-generation HTT-lowering approaches
- why boosting healthy HTT might help

And lots more!

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Much of the research we covered is only possible thanks to the generosity of HD families and their contributions to HD research, participating in clinical trials, observational studies, and selfless donations of samples and tissue. Were honored to share these results back with the community! #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

And that’s a wrap on the #HD2026 Milton Wexler Biennial Symposium! Thank you for sticking with us and we hope we have given you a flavour of how much HD research continues to evolve and grow.

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Certain fragments of HTT are more likely to form clumps in the nucleus than others. Since the nucleus contains DNA, Won-Seok thinks that they can interfere with processes, like gene regulation, including the chemical tags that regulate gene switches. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Won-Seok was also interested in understanding what happens to the shape and type of HTT clumps when the CAG number gets super-long, with more than 150 repeats. They can define different forms of HTT based on how it appears, moves, and dissolves in detergents. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Once again separating cells that have nuclear clumps (“inclusions” ) versus those that don’t, he showed that there are changes in which genes are turned on and off, and in the types of chemical “decorations” on the DNA and DNA packaging proteins. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Won-Seok is interested in the clumps of protein that show up in the nucleus of cells with CAG repeat expansions in HTT. He identified types of cells containing clumps in the nucleus, and found that they show higher levels of expansion than cells that don’t contain nuclear clumps. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

He has done work that separates this process into different phases across the life of a neuron with expanded CAG repeats, involving first slow and later very rapid expansion, all driven by somatic instability. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Won-Seok recaps recent discoveries about CAG repeat expansion: it seems to happen in vulnerable cells of the striatum and contribute to cell death in HD. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Our final speaker of the meeting is Won-Seok Lee, this year’s recipient of the Nancy S. Wexler Young Investigator Prize. His research is focused on how HTT clumps and somatic expansion can drive how neurons get sick in HD. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Now Vanessa is looking at the protein landscapes of specific proteins which are important in HD, including MED15, MSH3 and FAN1. Altogether, this is a wealth of data for the HD community - we look forward to hearing more from Vanessa as this project progresses. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

She also found that in HD, many of the proteins were in the wrong place. For example, some proteins which should be found on the boundary of the cell were instead found in the nucleus. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Vanessa’s maps allow us to dive into different changes observed in different compartments of a cell. An overall trend was a decrease in many important protein families, including those key to nerve cell signals, genetic message processing and gene regulation. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Vanessa is deploying these approaches in mice that model HD, to map out protein networks and landscapes in different compartments of both neurons and astrocytes. HD mice show many changes in these maps in comparison to control mice. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

To work out the location and amount of different proteins in the cell, she uses a chemical marking system that can determine when proteins are located near to each other. This marker system can be switched on and off and adapted to look in different tissues and at different timepoints. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Vanessa reminds us that when DNA is made into RNA messages, and messages are made into proteins, the RNA and protein levels don’t always correspond. Also, when most scientists study RNA or protein, they’re not able to look at their location within cells. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Vanessa Casha is up next. Her research maps how the amount and location of proteins within different types of cells can contribute to HD. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

The titanic effort that goes into understanding patterns within circuits, cells, and genes at this level of detail is astounding, and it’s particularly exciting when data collected at multiple levels points to similar pathways. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Cells with the most instability (longer CAG repeats) seem to lose their “identity,” with their genetic on/off states becoming less unique. Raleigh is looking in tons of detail at which genes and networks of genes might be driving this phenomenon. Some are already recognized as key to HD! #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Ralegh and colleagues have developed a way to link on/off changes in networks of genes with the amount of CAG repeat expansion and cell loss that is happening in single cells and brain areas. With this rich landscape of data the lab can create maps of vulnerability throughout the brain. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

He is once again reliant on precious samples of human tissue to look in high resolution at what’s happening in these subtypes of cells - work that would not be possible with the HD community. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Raleigh is digging even deeper into the question of why some cells get sicker than others in the HD brain, looking at sub-regions of the striatum and even specific types of cells. They get so specific that it’s necessary to re-define categories of cells among those affected most by HD. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Raleigh Linville is next to step up to the podium. Raleigh studies the striatum, the region of the HD brain that is most impacted by disease. He takes a comprehensive approach, termed multiomics in science lingo, synthesising diverse data on genetics, gene regulation and proteins. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Nat thinks that these chemical markers on the DNA could be exploited to develop both HD therapies and HD biomarkers. Turns out this is already being done in the cancer field, so maybe we can learn from their experiences to repurpose this idea for HD. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Nat’s model is that HTT interacts with gene regulating proteins, and this is disrupted in HD. Other enzymes regulating gene switches (chemical marks on the DNA itself) are also messed up, and all of this means that gene regulation is altered, even at the early stages of HD. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

While it might seem bad for the field that folks disagree on their data, this is actually great for constructive discussions to drive us forward to consensus. We need all these smart folks to butt heads (in a professional manner) so we can get to the ground truth! #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Nat thinks the early differences they observe are critical, but acknowledges that others think only the much later stages of this process matter in HD. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

One point of contention among HD scientists is in their beliefs about how messed up gene regulation needs to be in order to impact HD and drive disease. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

To better understand the gene regulation changes they have observed, Nat’s team looked at which gene switch signals are on and off. There are all kinds of signal changes that promote or suppress particular genes and they seem to occur mainly in the cells which get sickest in HD. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

One type of cell which doesn’t seem to show CAG repeat expansion in any context are glial cells. These cells provide support to neurons and are important for brain health in many ways. Glia have “rock solid” CAG numbers in every brain Nat’s team has analysed. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Interestingly, even cells with very long CAGs don’t necessarily survive better or worse than those with shorter CAGs, suggesting that CAG expansion is not the only thing that drives cell death in HD. #HD2026

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HDBuzz @hdbuzzfeed.bsky.social · 13/08/2026

Nat’s team are continuing to study brains selflessly donated by people with HD who have passed. His team have recently focussed on brain tissue from folks with Juvenile onset HD. They see expansions in most brain regions, and in more types of cells than are affected in adult onset HD. #HD2026

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