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Research articles

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HDBuzz is a collaboration of scientists who write Huntington’s disease research news in plain language for the global HD community.
They have allowed us to retrieve their articles for the NSW ACT Huntington’s community.


Global research collaboration

The Huntington Study Group (HSG), is the world’s first HD cooperative therapeutic research organization. Today, HSG is a world leader in facilitating high quality clinical research trials and studies that bring us closer to finding more effective treatments for HD and reducing the burden of HD for families affected by the disease.

HSG is an organization of compassionate professionals dedicated to finding treatments that make a difference, providing rigorous care initiatives, and improving the quality of life and outcomes for HD families. How? By bringing together families, medical professionals, clinical researchers, HD advocacy groups, and sponsors to raise awareness of HD, share knowledge and best practices, and develop innovative treatments.

Words from HSG website.

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Screening the entire genome for new drug targets for HD

Published date: 23 February, 2020

A recently published study in the journal ‘Neuron’ has identified new potential therapeutic targets for the treatment of Huntington’s disease (HD). The work conducted by Professor Myriam Heiman and colleagues used cutting-edge genetic technologies and discovered several genes could modify HD progression in their models in the lab. Many of these genes have not been ... Read more

The third dimension: using minibrains to understand brain development changes in HD

Published date: 6 February, 2020

A new publication used tiny 3D brain models created from human cells to show that the mutation that causes HD could lead to early changes in brain development. However, it’s clear that HD patients can, and do, develop fully mature brain cells that maintain normal function, in most cases, for decades. So let’s put these ... Read more

Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement

Published date: 3 January, 2020

DNA-based drugs called antisense oligonucleotides, or ASOs, are now in multiple clinical trials in Huntington’s disease, aiming to lower production of the harmful mutant huntingtin protein in the brain. Wave Life Sciences has been running parallel trials of two new ASO drugs, administered by injection into the spine. Just before the new year, Wave announced ... Read more

Huntington’s Disease research update

Published date: 12 November, 2019

Handwriting Can Help Identify Asymptomatic Huntington’s, Study Suggests.

Could molecular handcuffs lower the protein that causes Huntington's disease?

Published date: 12 November, 2019

Several approaches are being taken to lower the amount of the toxic huntingtin protein as a way to treat Huntington’s disease. Last week, a study reported a new strategy that helps target huntingtin for disposal by the cell. This approach is in its earliest stages and requires more testing, but the concept is sure to ... Read more

Exciting new Huntingtin lowering tool described

Published date: 5 August, 2019

An exciting new tool in the fight against Huntington’s disease has just been described. An international group of scientists have developed a new, targeted, way to lower levels of the mutant huntingtin protein. Huntingtin genetics: from gene to protein Huntington’s disease (HD) is caused by a genetic change – or mutation – in the DNA ... Read more

Details emerge of first Huntington’s disease gene therapy clinical trial

Published date: 17 July, 2019

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 nutshell AMT-130 is a huntingtin-lowering treatment, because ... Read more

Fresh Updates from First Huntingtin Lowering Study Publication

Published date: 7 May, 2019

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 this manuscript provides important new ... Read more

An Updated Trial Design for Roche's Huntingtin Lowering Trial

Published date: 21 March, 2019

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 lowering Readers of HDBuzz will be very familiar ... Read more

When interrupting is good: genetic hiccups that protect against Huntington's disease

Published date: 14 March, 2019

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 HD symptoms. What’s CAG and ... Read more