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At the recent Huntington’s Disease Society of America annual convention in Boston, UniQure announced crucial details of its planned clinical trial for its experimental therapy, AMT-130. We previously wrote about AMT-130 here, so this article covers the basics and what’s just been announced.Huntingtin-lowering gene therapy in an nutshellAMT-130 is a ... read more

Today saw the publication of the first manuscript describing a huntingtin lowering trial in Huntington's disease patients. This study, sponsored by Ionis and Roche, provided clear evidence of researchers’ ability to safely reduce mutant huntingtin protein in the spinal fluid. An overview of these results has previously been shared, but ... read more

Today we received word about a change to the design of Roche's ongoing huntingtin lowering trial - known as the GENERATION HD1 study. The update in the trial design is a surprise, but we believe it's a good thing. What exactly happened?The Roche approach to huntingtin loweringReaders of HDBuzz will ... read more

Multiple new studies have identified what may be the most important new fact about the genetics of Huntington's disease since the gene was discovered in 1993. At least two research groups around the world simultaneously report that a tiny genetic hiccup in the HD gene has a big impact on ... read more

Jeff and Ed report from the Huntington’s Disease Therapeutics Conference - the biggest annual gathering of HD researchers. This year’s conference is bigger and more exciting than ever.Read About Day 1 here.Read about Day 2 here.Thursday morning - Huntingtin structure & functionThe final day of the HD Therapeutics Conference begins ... read more

Jeff and Ed report from the Huntington’s Disease Therapeutics Conference - the biggest annual gathering of HD researchers. This year’s conference is bigger and more exciting than ever.Read about Day 1 here.Advanced tools for translational researchMorning everyone! The second day of the 2019 HD Therapeutics Conference is kicking off in ... read more

Jeff and Ed report from the Huntington’s Disease Therapeutics Conference - the biggest annual gathering of HD researchers. This year’s conference is bigger and more exciting than ever.Tuesday morning - white matterGood morning from sunny Palm Springs, and the first day of the 2019 Huntington's Disease Therapeutics Conference! Up first ... read more

Dutch-American company uniQure has received approval from the US drug regulator the FDA, to begin the first ever gene therapy trial in Huntington's Disease. Their plan is to use a virus, injected into the brain, to turn cells into a factory that makes a weapon to lower the harmful huntingtin ... read more

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 ... read more

We've had a run of exciting updates from Ionis and Roche/Genentech about their program to test a drug that lowers production of the huntingtin protein – but they're no longer the only game in town. Recently several other players - including Wave Life Sciences, PTC Therapeutics, and Voyager Therapeutics, have ... read more

On Wednesday, September 26th, HDSA hosted a webinar presented by Genentech/Roche in which representatives from their Huntington’s disease team shared details and answered questions about upcoming Genentech/Roche clinical trials for HD. HDSA received hundreds of questions before, during, and after the webinar. Here HDSA's staff scientists, with guidance from Genentech/Roche, ... read more

Juvenile-onset HD is really rare, but a new study has helped us understand what the symptoms are and how they change over time. This is really important and in time will help us work out whether huntingtin-lowering and other treatments work in kids.What is juvenile Huntington's disease?When symptoms of Huntington's ... read more

It's a time of huge developments in the field of huntingtin-lowering drugs, which aim to reduce production of the huntingtin protein that causes Huntington's disease. At the European Huntington's Disease Network (EHDN) meeting this past week, Roche announced firm details of the first ever pivotal study of a huntingtin lowering ... read more

The LEGATO-HD trial of laquinimod to slow progression of Huntington's disease did not meet its primary endpoint. What's an endpoint, and what does this all mean for HD families?What was the Legato-HD trial all about?The Legato-HD trial was designed to test whether treatment with a drug called Laquinimod could slow ... read more

The Enroll-HD study is the largest clinical study in Huntington's Disease ever. What's it all about, and why is Enroll-HD so important in the fight against HD? Join HDBuzz's own Jeff Carroll as he joins the Enroll-HD study to learn more.What is observational research?In some ways, HD is very simple ... read more

Exciting news for the HD research field this month as a new genetically faithful model has been created in pigs. Using a combination of cutting edge DNA editing tools researchers were able to generate HD pigs that not only replicated the selective brain cell death seen in HD, but also ... read more

Recently the Critical Path Institute announced a new effort - the Huntington's Disease Regulatory Science Consortium, or HD-RSC. This collaboration with many international partners aims to speed the development of new HD treatments. What's all this about, and how could it help HD patients?What is C-Path?The Critical Path Institute is ... read more

Earlier this week, Ionis Pharmaceuticals put out a press release after presenting data from the HTTRx (RG6042) Huntingtin-lowering clinical trial program at a scientific conference. Ionis’ partner Roche also released a community statement. Our friend at Huntington’s Disease Society of America, Dr Leora Fox, has prepared this Q&A in response ... read more

Drs Rachel Harding and Tam Maiuri are taking a leap of faith to make discoveries in Huntington’s Disease (HD) research swift and open to all. They are publishing their lab notes on fundamental HD research online in real time, at the risk of losing credit for their work. As a ... read more

The cause of Huntington’s disease has been known since 1993, but the physical structure of the healthy huntingtin protein proved difficult to discover until now. German scientists have now revealed the shape of the huntingtin protein for the first time. Although the structure of the mutant form of the protein ... read more

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 Trials from both Wave Life Sciences and Ionis Pharmaceuticals.Thursday morning - huntingtin proteinEvery HD patient has inherited ... read more

Updates from day 2 of the Huntington's Disease Therapeutics Conference focusing on DNA repair in HD.Wednesday morning - DNA repair in HDGood morning from the 2018 HD Therapeutics Conference! Today's update is relatively brief because the afternoon was focused on poster presentations. The morning session focused on the role of ... read more

Jeff and Ed report from the Huntington's Disease Therapeutics Conference - the biggest annual gathering of HD researchers. This year's conference is bigger and more exciting than ever.Tuesday morning - brain connectionsGood morning from the first day of the 2018 HD Therapeutics Conference in sunny Palm Springs!Rui Costa, Columbia University, ... read more

New hints are emerging about the normal role of the gene that causes Huntington's disease. A recent report uses cutting edge techniques to study this question in cells growing in the lab. We'll help separate the fascinating new science from some scary-sounding headlines. Cool science, shame about the reportingYou might ... read more

A recent mouse study of a drug known as CTEP suggests the drug is surprisingly helpful for HD-like symptoms in mice. This is a welcome surprise, because it suggests a well-understood brain process might be a useful drug target for future HD research. Busy as a brain cellHuntington's Disease is ... read more

The research news, announced on 11 December 2017, that a research team from the Huntington's Disease Centre at University College London have made significant step towards a possible treatment for Huntington's disease, raised many questions for the Huntington's disease community. Dr Ed Wild answered some of these questions on behalf ... read more

In an announcement likely to stand as one of the biggest breakthroughs in Huntington's disease since the discovery of the HD gene in 1993, Ionis and Roche today announced that the first human trial of a huntingtin-lowering drug, IONIS-HTTRx, demonstrates that it reduces mutant huntingtin in the nervous system, and ... read more

Genome editing is a much-discussed frontier in medical science at the moment, with ‘DNA surgery’ having the potential to treat or cure genetic diseases like Huntington’s. Here we look at what this technology can currently do and discuss the challenges that still lie in the way. We’ll also discuss how ... read more

A new analysis of clinical data from the TRACK-HD and COHORT studies proposes a way to design of clinical trials designed to delay the onset of HD, rather than treating symptoms after they occur. What's the history of clinical trials in HD?Clinical trials are experiments conducted to see whether a ... read more

Some techniques aimed at lowering mutant huntingtin can also affect the normal form of the protein. With clinical trials underway, it’s all the more important to understand the role of normal huntingtin in the adult brain. Researchers recently inactivated the huntingtin gene in healthy adult mice of different ages. They ... read more

A new exciting chapter in Huntington’s disease (HD) treatment is just beginning – WAVE Life Sciences have announced PRECISION-HD1 and 2, clinical trials of two new drugs that lower the mutant Huntington’s disease protein. We’re excited about this novel approach to huntingtin lowering therapies, but these are early days and ... read more

CRISPR-Cas9 is an experimental gene editing technique used to make precise changes in DNA. For the first time, scientists have used this approach to attack the Huntington's Disease mutation in the brain cells of a mouse. Other researchers are refining CRISPR-Cas9 to be more efficient, specific, and safe. It’s still ... read more

Ionis Pharmaceuticals launched the first ever trial of a huntingtin-lowering drug – sometimes called a 'gene silencing drug' – in late 2015. In a significant update, the company has announced two important milestones: the trial is now fully recruited, and an 'open-label extension' will be activated for the volunteers in ... read more

What if a blood test could provide information about the status and course of HD? This is the premise of seeking HD biomarkers; they may someday help guide treatment decisions and predict how symptoms will change. A team of researchers spanning multiple countries recently analyzed blood, brain images, and clinical ... read more

If media reports of a "wonder-drug" that could "stop all neurodegenerative brain diseases, including dementia” seem too good to be true, that's because they are. The truth behind the headlines is that researchers tested thousands of already-licensed drugs in worms, and a couple that went on to show beneficial effects ... read more

The final day of the 2017 Huntington's Disease Therapeutics conference brought updates on research to better understand how HD affects brains, and breakthroughs in using brain cells to understand and treat HD.Connections in the brainAndrew Leuchter from UCLA works on ways to study the brain's electrical activity to monitor HD. ... read more

Day two of the conference looks at some of the most promising approaches to fighting Huntington's disease.Huntingtin lowering therapiesExciting session planned for this morning, as the conference discusses "Huntingtin lowering" approaches to treating HD.The first talk is from HDBuzz's own Ed Wild (University College London), who is interested in developing ... read more

Hi everyone! It's the annual Huntington's Disease Therapeutics Conference in Malta. Around 350 scientists from round the world all working on HD are gathered here to discuss the latest research developments. We're reporting live via twitter and aggregating our updates into daily articles.Big news from big dataThe first speaker is ... read more

Exciting new studies provide evidence that a particular kind of cellular trafficking goes awry in Huntington's Disease. Specifically, researchers have learned that traffic in and out of the cells control center - the nucleus - breaks down in HD. These findings open up new avenues for HD research.Cellular border controlAt ... read more

Significant news for the Huntington's disease community this week, as the USA's drug regulator, the FDA, has formally approved Austedo, also known as deutetrabenazine, for prescribing in HD. This modified form of tetrabenazine helps control chorea, the jerky movements often found in HD patients, but is taken twice rather than ... read more

Beyond affecting movement, mood, and thinking, HD involves a complex set of changes to the body that can be difficult to predict in different individuals. Recently, researchers were able to identify consistent early changes in metabolism by studying a group of HD sheep. This large animal model is helping scientists ... read more

Cannabis, or medical marijuana, has been touted as a treatment for lots of conditions, and Huntington’s disease is no exception. Whenever it hits the news there’s a lot of interest, and recently cannabis found the spotlight again with videos claiming it can reverse the nerve cell damage in Huntington’s disease. ... read more

Cannabis, or medical marijuana, has been touted as a treatment for lots of conditions, and Huntington’s disease is no exception. Whenever it hits the news there’s a lot of interest, and recently cannabis found the spotlight again with videos claiming it can reverse the nerve cell damage in Huntington’s disease. ... read more

DNA damage is a hot topic in HD – and new research offers an intriguing explanation. Canadian researchers have uncovered a potential role for huntingtin in the repair of DNA. They speculate that the normal protein is recruited to the nucleus to provide a supporting scaffold for a construction crew ... read more

DNA damage is a hot topic in HD – and new research offers an intriguing explanation. Canadian researchers have uncovered a potential role for huntingtin in the repair of DNA. They speculate that the normal protein is recruited to the nucleus to provide a supporting scaffold for a construction crew ... read more

Huntingtin, the protein responsible for Huntington’s disease, is fundamentally important for fetuses to develop in the womb, but we don’t know yet exactly what part it plays in this intricate process. Normally, neurons start life deep within the developing brain, migrate out to the surface and then make a network ... read more

Huntingtin, the protein responsible for Huntington’s disease, is fundamentally important for fetuses to develop in the womb, but we don’t know yet exactly what part it plays in this intricate process. Normally, neurons start life deep within the developing brain, migrate out to the surface and then make a network ... read more

Researchers have long known that patients with a greater number of CAG repeats in their HD gene tend to display motor symptoms sooner than those with fewer CAG repeats. However, this relationship is not perfect. Sometimes HD patients display symptoms at a drastically different time than expected based on the ... read more

Researchers have long known that patients with a greater number of CAG repeats in their HD gene tend to display motor symptoms sooner than those with fewer CAG repeats. However, this relationship is not perfect. Sometimes HD patients display symptoms at a drastically different time than expected based on the ... read more

Researchers have long believed that the Huntington's disease gene causes problems by telling cells to make a harmful protein. Intriguing new animal work from researchers in Spain suggests we might want to look at more than one suspect to completely fix the problems caused by the HD mutation.The central dogma ... read more