Huntingtin-lowering is an HD treatment strategy that usually involves targeting the HD gene itself, but some researchers are exploring roundabout ways to achieve the same effect. A research team in China recently developed a drug to target a protein called GPR52, and tested its ability to lower huntingtin in mouse models of HD and in cells. They published their work recently, and HDBuzz covered the story this week. “Small molecule” strategies like this are in the early stages of development, but are still intriguing, because they could potentially be delivered less invasively than genetic drugs.
HD Insights Podcast
The Huntington Study Group (HSG) conducts long-form interviews with individuals making an impact on HD
research and care. In the latest episode, Dr. Michael Hayden, Chief Executive Officer for Prilenia Therapeutics, discussed the global PROOF-HD clinical trial, which was also presented in a recent HDSA webinar. PROOF-HD is testing the effectiveness and safety of a drug called pridopidine in patients with early stage HD. From HSG: “Dr. Hayden spoke about this new study’s unique endpoint for total functional capacity. You will also want to hear his inspiring personal journey that led him into Huntington disease research growing up in South Africa.”
Survey Study: Attitudes Towards HD Genetic Testing
A team of researchers at the London Business School have created a study aimed at understanding people’s attitudes toward the difficult and complex decision of whether to test for Huntington’s disease. The purpose of this study is to understand people’s attitudes toward testing for HD and the psychological consequences of testing. The findings of this study could potentially help complement consulting procedure for people who have or are at risk of developing Huntington’s disease or have already developed Huntington’s disease.
The research study is organized by Professor Simona Botti and PhD candidates Nazli Gurdamar Okutur and Selin Goksel at London Business School. The study consists of filling in a short online survey that takes approximately 10 minutes.
The researchers are looking for:
• People who have Huntington’s disease
• People who have tested positive but are not yet showing symptoms
• People who are at risk but haven’t tested
• People who have someone they care about who is affected by HD
If you are 18 years of age or older and are interested in participating, please click on this link: http://lbs.eu.qualtrics.com/jfe/form/SV_9zqupfDREDm2D1H