Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.

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Update confirms Huntington's disease 'gene silencing' trial on track

Update confirms Huntington's disease 'gene silencing' trial on track

Dr Jeff Carroll on June 22, 2017

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 the current trial. While nothing is guaranteed, this bodes well for the future of this important program.

New study reveals a potential HD biomarker

New study reveals a potential HD biomarker

Leora Fox on June 07, 2017

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 exams from the TRACK-HD study. They found that blood levels of a protein called neurofilament light chain corresponded with the severity of HD, making it a potential biomarker.

Has a "wonder drug" for dementia been discovered? (Spoiler alert: no.)

Has a "wonder drug" for dementia been discovered? (Spoiler alert: no.)

Dr Michael Flower on May 07, 2017

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 in mouse models of two rare forms of dementia. While it gives researchers two new leads, this research doesn't prove anything about these drugs in patients with neurodegenerative diseases, and has virtually nothing to do with Huntington's disease at all.

Huntington's Disease Therapeutics Conference 2017 - Day 3

Huntington's Disease Therapeutics Conference 2017 - Day 3

Dr Ed Wild on April 30, 2017

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.

Huntington's Disease Therapeutics Conference 2017 - Day 2

Huntington's Disease Therapeutics Conference 2017 - Day 2

Dr Jeff Carroll on April 29, 2017

Day two of the conference looks at some of the most promising approaches to fighting Huntington's disease.

Huntington's Disease Therapeutics Conference 2017 - Day 1

Huntington's Disease Therapeutics Conference 2017 - Day 1

Dr Jeff Carroll on April 27, 2017

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.

Enemy at the gates – huntingtin disrupts nuclear transport

Enemy at the gates – huntingtin disrupts nuclear transport

Tom Peskett on April 21, 2017

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.

FDA approves a new drug for symptoms of Huntington's disease

FDA approves a new drug for symptoms of Huntington's disease

Dr Jeff Carroll on April 05, 2017

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 three times a day.

Sheep leading the flock: metabolism and biomarkers in HD

Sheep leading the flock: metabolism and biomarkers in HD

Leora Fox on March 27, 2017

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 to track altered substances in the blood that could predict HD progression and response to treatment.