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Welcome to HDBuzz! This special page is for people who are new to Huntington’s disease, or new to the world of HD research.

Reading the articles linked here will help you pick up the basics of what Huntington’s disease is, and get up to speed with some of the most promising things scientists are doing to come up with effective treatments for HD.

Huntington’s disease – the bare essentials


  • Our Science FAQ covers the very basics of HD and introduces some ideas about why we need research to find treatments.
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About HDBuzz


  • Our HDBuzz FAQ and People Page explain what HDBuzz is, and the people behind it.
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  • And our Funding page explains where we get our money from and how we make sure HDBuzz is neutral and reliable.
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The most promising possible treatments

Some of the world’s top scientists are working round the clock to develop treatments for Huntington’s disease – and real progress is being made. Dozens of possible treatments are being worked on, and every day a successful treatment gets one day closer. Here are our personal top four approaches.


  • Huntingtin lowering. Huntington’s disease is caused by a faulty protein, and huntingtin lowering drugs tell cells to make less of that protein. Huntingtin lowering is sometimes called “gene silencing”. Right now it is our best hope for an effective treatment. Read about it in our Gene Silencing Primer. In December 2017 the first successful huntingtin-lowering trial was announced. An “ASO” drug called HTTRx, made from artificial DNA, was injected into the spinal fluid of 46 volunteers 4 times each over 3 months. The drug successfully lowered the level of the mutant protein in the fluid, and was safe and well tolerated. A larger, longer trial called Generation-HD1 will test whether the drug, now renamed RG6042, can slow the progression of HD.
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  • Several other approaches to Huntington-lowering are being developed and tested, including other ASO drugs, viruses containing instructions to make less of the huntingtin protein, and even “small molecules” that could be taken as a pill.

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  • KMO inhibition. KMO is a chemical machine that determines the balance of certain helpful and harmful chemicals in cells. Our article on KMO Inhibitor Drugs explains how blocking it might be useful for treating Huntington’s disease.

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  • Protein tagging. Scientists call it post-translational modification, we call it tagging. Our cells add little chemical tags to proteins, and the tags alter the proteins' behavior. One approach to treating Huntington’s disease is altering the way cells tag the harmful protein. Read all about it in our article on Re-routing the Huntingtin Protein then you can read our latest articles on tagging.

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Living with HD

Some of our most popular articles offer the latest information about day-to-day issues faced by Huntington’s disease family members.


  • ‘Making Babies’ explains how people at risk of HD can use assisted fertility methods to have HD-free kids – even if the would-be parents don’t want to be tested themselves.
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  • Our article on the ‘Genetic Gray Area’ of HD sheds light on the often confusing topic of people whose HD genetic test result isn’t quite positive or negative, but somewhere in the middle.
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  • Many HD-affected people are frustrated by a lack of interest or expertise from care professionals. Take a look at our article on Closing the Care Gap to find out about the expert guidance available to professionals – why not show it to the professionals looking after you?
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Taking action

Successful treatments to prevent, slow, reverse or cure Huntington’s disease will arrive more quickly if more people help out. Here’s what we recommend:


  • Sign up for Enroll-HD, the largest global study of HD family members. Anyone from an HD family can sign up and you don’t need to have had a genetic test to do so. A short annual assessment involving questionnaires, thinking tests and a blood sample is all it takes to help us understand HD and put you in pole position for clinical trials.
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  • In the USA, look at the HDSA’s TrialFinder, which will tell you what drug trials and other research studies are going on near you.
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  • In Canada, look at the HSC’s Clinical Trial Locations page.
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  • In Europe, check out the Euro-HD Network.
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  • Elsewhere, the International Huntington Association will help you get involved.
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Getting behind the headlines

A key mission of HDBuzz is to help our readers sort out the hope from the hype. News and blog stories can sometimes give a false impression of how promising a particular treatment is, or how soon it could deliver for HD-affected people.


  • Our Ten Golden Rules article suggests ten simple steps to help you draw hope from a science news story, without being disappointed by impossible promises.
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Meanwhile, these articles deal with areas of HD research that generate a lot of headlines but – while interesting and potentially useful – aren’t necessarily able to live up to the hype.


  • Mesenchymal Stem Cells (stem cells from bone marrow) – an interesting approach but far from being a treatment for HD right now.
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  • Our Stem Cell Primer explains the real value of stem cells, right now – as tools to help us understand Huntington’s disease.
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  • Pridopidine – an experimental drug aimed at improving movements in HD – isn’t approved for human use because clinical trials haven’t shown it works.
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The very latest

We regularly produce reports from major scientific meetings and conferences. They’re a great way to get an overview of all the hottest Huntington’s disease research. Catch up on the latest conference news here.

Make yourself at home!

HDBuzz is designed so that each article makes sense on its own and gives you all the background info you need. So don’t be afraid to explore.


  • Each article has a learn more box, containing links to original sources and background information.
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  • Every article has a topics box so you can easily find content on a particular subject.
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If there’s something about HD research you’d like explained but can’t find here, feel free to use the ‘Suggest an article’ box on the front page.

And to make sure you don’t miss anything exciting, follow us on Twitter or Facebook, or sign up for Email updates

Welcome aboard. We’re glad you found us.

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Latest Research Articles

Treatment for neurological disorder could be repurposed for Huntington’s disease patients

Published date: 22 October, 2020

While developing a drug called branaplam for patients with SMA, the pharmaceutical company Novartis discovered that it could hold promise for people with HD. The FDA has granted a special status called Orphan Drug Designation to branaplam. An existing drug…for huntingtin lowering? The pharmaceutical company Novartis has announced that the U.S. Food and Drug Administration ... Read more Treatment for neurological disorder could be repurposed for Huntington’s disease patients

Updates from the EHDN Plenary Meeting 2020

Published date: 9 October, 2020

In September, the European Huntington’s Disease Network (EHDN) hosted a virtual webinar event which comprised presentations on some of the latest scientific research as well as clinical studies of Huntington’s disease (HD). Researchers, doctors, patients and other interested folks, tuned in for an afternoon of talks as well as question and answer sessions to learn ... Read more Updates from the EHDN Plenary Meeting 2020

Sad news from the SIGNAL study: pepinemab does not influence HD symptoms

Published date: 23 September, 2020

The SIGNAL clinical trial was designed to test a drug called pepinemab in people with early Huntington’s disease. The key results of that trial were recently announced, and unfortunately, pepinemab did not slow or improve HD symptoms as hoped. What was the SIGNAL trial, and who participated? The SIGNAL trial was launched in 2015 by ... Read more Sad news from the SIGNAL study: pepinemab does not influence HD symptoms

When genes are unstable: targeting somatic instability in HD

Published date: 8 September, 2020

What is somatic instability? We tend to think of DNA as a fixed blueprint, an overarching plan for the biological bricks and bridges that constitute our cells, organs, and bodies. But like any good plan, DNA is actually dynamic and adaptable. It gets frequent use as a template for creating the RNA messages that pave ... Read more When genes are unstable: targeting somatic instability in HD

Working as a team: Changes in brain development mean some brain regions may be slacking off

Published date: 17 August, 2020

The effect of the HD genetic expansion on brain development has been a hot topic in HD research. A team of researchers led by Dr. Sandrine Humbert at the Grenoble Institut Neurosciences, examined human fetal tissue to show that the mutant HD gene causes very early changes in the patterns of early brain development. But ... Read more Working as a team: Changes in brain development mean some brain regions may be slacking off

Caution urged for the use of gene-editing technology CRISPR

Published date: 12 August, 2020

A gene-editing tool known as CRISPR has been heralded as a breakthrough technology for scientists in the lab but also as a potential strategy to treat numerous genetic diseases, including Huntington’s. But a series of recent studies has suggested that CRISPR is less precise than previously thought, leading to unintended changes in the genome. Three ... Read more Caution urged for the use of gene-editing technology CRISPR

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