Research articles

Click on this link to read our latest research articles.

Click on this link to find out more about Huntington’s NSW ACT Research grant opportunities.

HD Buzz

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.

Westmead HD Outreach Service

The Outreach Service is part of a multidisciplinary team of medical, nursing and allied health services which provides review, intervention, treatment, case management, counselling, support, education and management of problems and issues associated with Huntington’s for patients, their families and carers. In addition to this care, the service has been involved in local and major international research studies since its establishment at Westmead Hospital in 1995. Hundreds of volunteers have generously participated in research to better understand the disease and in the search for effective treatments.

Westmead Hospital is a HSG Credential Research Site. Professor Clement Loy is the Principal Investigator for this site.

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. They bring 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.

Enroll-HD

Enroll-HD is a clinical research platform and the world’s largest observational study for Huntington’s disease families. It is a resource for the entire HD community, including families, clinicians, researchers, advocates, and anyone else who has a connection to or an interest in HD.

Huntington’s Disease Network of Australia

The Huntington’s Disease Network of Australia (HDNA) is a project conceived by Professor Julie Stout of Monash University. Formed in 2020 to coordinate efforts that enhance care and services for HD and prepare for the advent of new HD treatments. The Map-HD Registry is for people in Australia who are affected by HD. All family members or people affected by HD are encouraged to register, whether or not they are at risk themselves.

Oral drug may change the story for huntingtin lowering

Published date: 1 February, 2022

Huntingtin lowering has gained lots of attention in HD research, and for good reason. It was the first potential treatment designed to directly target the cause of HD – the huntingtin protein. But there are limitations to current huntingtin lowering approaches: they require delivery to the spinal fluid or brain surgery for delivery, can show ... Read more

Finding the silver lining: an update on the Roche GENERATION-HD1 trial data

Published date: 21 January, 2022

On January 20th, Roche shared a set of long-awaited data from the GENERATION-HD1 trial of tominersen, a huntingtin-lowering drug. Although the trial did not meet its key goals, and too-frequent dosing may even have made patients worse, new findings have given us some cause to hope that tominersen could still benefit certain people with HD. ... Read more

KINECT-HD trial shows valbenazine improves involuntary movements in Huntington's disease

Published date: 14 December, 2021

Chorea – unwanted fidgety or jerky movements – is one of the most noticeable features of Huntington’s disease (HD). There are already drugs to treat chorea licensed specifically for use in HD, as well as so-called off-label treatment options. But all the existing treatments have potential downsides, so new drugs to reduce chorea could be ... Read more

“Seeing” the toxic huntingtin protein in people with HD

Published date: 8 December, 2021

Scientists have developed a tool which allows us to “see” the toxic clumps of the huntingtin protein using special scanners. People with Huntington’s disease (HD) make a toxic form of the huntingtin protein which forms clumps in cells of their bodies, which accumulate during HD progression. Tracking how these clumps form over time in people ... Read more

Updates from the EHDN Meeting 2021

Published date: 28 October, 2021

Last month, the Huntington’s disease (HD) research community, patients and other stakeholders met online at the European Huntington’s disease network (EHDN) conference. Despite the ongoing global pandemic, there is a tremendous amount of work underway in labs and clinics around the world as researchers continue to better understand HD and how we might best treat ... Read more

Real talk: Q&A with Roche about GENERATION-HD1

Published date: 28 September, 2021

At the end of day 1 of the European Huntington’s Disease Network (EHDN) conference, the HDBuzz team (minus one) sat down for a zoom chat with the team at Roche to have a frank, candid discussion about the recent halting of GENERATION-HD1, the Phase 3 clinical trial that was testing the ability of the antisense ... Read more

Another tool in the box: Creation of a molecular “dimmer switch” advances gene editing

Published date: 30 August, 2021

A team of scientists recently created an innovative genetic system where a drug taken by mouth could be used to control the action of a gene editor, like those used in CRISPR systems. This has useful applications for research studies in cells and animals, and perhaps most importantly, could lead to improvements in the safety ... Read more

Unpacking recent gene therapy press

Published date: 16 August, 2021

A recent announcement from Voyager Therapeutics outlined a shift in the company’s strategy towards an exciting new technology for gene therapy delivery. Unfortunately this also means that in the short term, they have dropped previous plans to test an HD gene therapy in people with HD. While this news is disappointing, the decision to embrace ... Read more

Does blood hold the key to testing treatments earlier in HD patients?

Published date: 4 August, 2021

Researchers at Johns Hopkins led by Wenzhen Duan have developed a non-invasive way to track progression of Huntington’s disease (HD) which could be used before patients even start showing symptoms. Using a type of brain scan called an MRI, the researchers have shown that in mouse models of HD they can accurately measure the amount ... Read more

A first for CRISPR gene editing could have wider applications for human disease

Published date: 21 July, 2021

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 other brain disorders. CRISPR-Cas9 Clustered ... Read more

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Research articles

HD Buzz

Westmead HD Outreach Service

Enroll-HD

Huntington’s Disease Network of Australia

Oral drug may change the story for huntingtin lowering

Finding the silver lining: an update on the Roche GENERATION-HD1 trial data

KINECT-HD trial shows valbenazine improves involuntary movements in Huntington's disease

“Seeing” the toxic huntingtin protein in people with HD

Updates from the EHDN Meeting 2021

Real talk: Q&A with Roche about GENERATION-HD1

Another tool in the box: Creation of a molecular “dimmer switch” advances gene editing

Unpacking recent gene therapy press

Does blood hold the key to testing treatments earlier in HD patients?

A first for CRISPR gene editing could have wider applications for human disease

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