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A recent clinical trial successfully tested the safety of CRISPR gene editing to reduce the amount of a toxic protein in patients with Familial Transthyretic (TTR) Amyloidosis. Although this study is unrelated to Huntington’s disease, it’s a first for gene editing, and the results could have implications for HD and ... read more

Pridopidine is a drug developed to treat Huntington’s disease (HD) and now scientists have a clearer understanding of how it works in the body and brain. In a series of academic papers, researchers figured out that pridopidine is working by targeting a particular receptor protein called S1R. With this new ... read more

We are back for the last day of the virtual 2021 CHDI Therapeutics conference. This article summarises our live Twitter updates on the exciting science being presented, which you can find with the hashtag #HDTC2021. The final session of the conference will provide the latest news on more Huntington’s disease ... read more

We are back with Day 2 of the virtual 2021 CHDI Therapeutics conference. This article summarises our live Twitter updates on the exciting science being presented, which you can continue to follow with the hashtag #HDTC2021. The morning session focussed on promising HD therapeutics that are in preclinical development and ... read more

The CHDI Therapeutics Conference 2021 kicked off today. This article summarizes our live Twitter updates on the exciting science being presented, which you can continue to follow with the hashtag #HDTC2021. The morning of Day 1 focused on clinical trial updates, while the afternoon explored genetic modifiers of HD and ... read more

It seems like the HD community has been inundated with updates from different companies and clinical trials recently. The news is far from complete doom and gloom; although there have been some real disappointments from some of the frontrunner trials, there are also positive updates from many different companies and ... read more

Two recent press releases from uniQure provide welcome good news: the first ever HD gene therapy, known as AMT-130, has been administered via brain surgery to a small set of participants in an early safety trial. At the same time, uniQure has published findings in HD animal models that increase ... read more

The Huntington’s Disease community faced more disappointing news this week as Wave Life Sciences shared that they will discontinue development of two ASOs they had been testing in people with HD. Designed to decrease harmful huntingtin protein while keeping the healthy form intact, these experimental therapies unfortunately did not have ... read more

Very sad news was announced today as Roche and Ionis declared that the large ASO study they're running in Huntington's disease patients has been halted early. Importantly, no specific new safety concerns were raised so far, but nevertheless dosing of the study drug, tominersen, as well as the placebo, has ... read more

Families around the world are being impacted by COVID-19, but hope is on the horizon in the form of revolutionary new vaccines which were developed in record time. How is the coronavirus impacting HD families, and should they be worried about any of the vaccines coming to market? Should HD ... read more

Scientists have found new leads for how to reduce the levels of the Huntington’s disease (HD) protein by targeting a protein called GPR52. A team of researchers working in Shanghai, China developed small drug-like molecules which lower huntingtin protein levels in HD tissue culture models and in HD mouse models. ... read more

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 ... read more

A gene known as 'MSH3', which encodes a protein involved in fixing and maintaining our DNA, has become a hot topic in Huntington’s research since being implicated as a key driver of the disease by multiple genetic studies. In a recent publication, a team of scientists from the National University ... read more

The second day of the HSG conference was another busy day of presentations from HD researchers and clinicians. The day kicked off with a talk from Vaccinex who gave us an overview of their work on the SIGNAL clinical trial. Unfortunately, pepinemab, the medicine tested in this trial, did not ... read more

The Huntington Study Group (HSG) is a clinical research network focused exclusively on HD. Yesterday the HSG annual conference began with a schedule jam-packed with virtual talks from researchers, clinicians and different companies who are all working towards finding new medicines for HD. The day encompassed many interesting presentations which ... read more

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. ... read more

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 ... read more

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 ... read more

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 ... read more

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 ... read more

Dopamine is an important chemical messenger in the brain that becomes imbalanced in Huntington’s disease. Researchers recently described a creative way to restore the balance and treat symptoms in HD mice, using an antihistamine drug that acts on hybrid dopamine receptors. It’s an innovative approach to HD therapeutics, but don’t ... read more

Researchers have known for quite some time that HD causes a progressive loss of neurons. But what if we could find a way to fill their place? In a new report, researchers used an intriguing strategy in living mice to do just that – they converted a different type of ... read more

A new study headed up by Dr. Sarah Tabrizi, a pioneer in HD research, assessed pre-manifest HD young adults many years from predicted symptom onset with a battery of clinical tests. The goal of this study was to identify a sweet spot – a time when HD participants weren’t experiencing ... read more

A team of scientists has recently published their findings on how our bodies are able to repair brain and spinal cord injuries. They found that the huntingtin protein plays an important role in repairing damaged nerve cells. Repairing nervous system damage – the holy grail of medical scienceIt has long ... read more

A collaborative team of scientists from Canada and Japan have identified a small molecule which can change the CAG-repeat length in different lab models of Huntington's disease. CAG repeats are unstableHuntington’s disease is caused by a stretch of C, A and G chemical letters in the Huntingtin gene, which are ... read more

COVID-19, short for coronavirus disease 2019, has taken the world by storm in almost every sense – many people have been infected with the SARS-CoV-2 virus, it’s created shopping pandemonium in stores, and many people are isolated at home. But behind that frenzied storm, scientists around the world have been ... read more

Our new writers Rachel Harding and Sarah Hernandez report from the Huntington’s Disease Therapeutics Conference - the biggest annual gathering of HD researchers.Tuesday morning - HD genotype and phenotypeGood morning from sunny Palm Springs! We’re excited to be here for the 15th Annual HD Therapeutics Conference. This year in addition ... read more

Rachel and Sarah report from the Huntington’s Disease Therapeutics Conference - the biggest annual gathering of HD researchers.Be sure to catch up on Day 1 and Day 2.Thursday morning - Huntingtin loweringGood morning everyone! We are back for Day 3 at the CHDI meeting in Palm Springs which is all ... read more