As we mentioned in January, we’ll be hosting an HD Research Year in Review webinar on Thursday, February 18th, at 12 PM Eastern, to talk about the extraordinary progress made in the HD research field this year. Please join us to learn more about the triumphs of 2020 at HDSA and beyond in spite of the difficult circumstances.
HDSA also recently released our 2020 Year In Review Magazine that talks about all of the activities that continued to support HD families and HD research during and in spite of the pandemic. Check it out here!
uniQure Press Release: Study cleared to move forward
This week the gene therapy company uniQure shared exciting news in a press release announcing that their trial of AMT-130 has been cleared to move forward. The drug is a huntingtin-lowering therapy delivered via a single brain surgery, and it is currently being tested in a small safety trial. This trial is proceeding slowly and cautiously because it is an unprecedented approach to treating HD. Four patients have already undergone the surgery, and after review of six-month and three-month data, there have been no major safety concerns. The next step is to enroll six additional patients, and these ten will form the first main trial group (known as a cohort). We’re excited about the progress of the first gene therapy trial for HD and hope to hear more by the end of this year.
Help Researchers Develop HD Clinical Trials Education
If you’ve got 25 minutes to spare and want to help researchers develop educational materials about HD clinical trials, consider participating in a research project conducted by the MGH Institute of Health Professions’ Genetic Counseling Program. You’ll be asked to watch a video and complete a survey, and can enter for a chance to win a $25 Amazon gift card. For more information about the project and the research team, view the study flyer or go directly to the study page.
Special Issue of the Journal of Huntington’s Disease
The Journal of Huntington’s Disease is an international academic journal dedicated to facilitating progress in understanding HD and developing treatments. The latest installment is open access, meaning that it is available to anyone to read, and it is a Special Issue on the topic of DNA repair and somatic repeat expansion in HD. Somatic repeat expansion is the phenomenon of CAG repeats getting longer over time in some cells within a person’s body. The study of this topic is important for understanding why some people get HD so much earlier than others, and how we can combat this to delay age of onset.
The Special Issue Review editors note: “This exciting issue covers topics ranging from the discovery of somatic repeat expansion in HD, to our current understanding of the molecular mechanisms involved, and the development of potential new therapies targeting these mechanisms.”
Although the articles in the Journal of Huntington’s Disease are directed towards scientists, you may enjoy checking them out or browsing through to get a sense of the hot topics within this sub-field of HD research.