HDSA received hundreds of questions during and after the September 26th webinar presented by Genentech/Roche representatives about upcoming clinical trials. We thank you for your patience as we prepared responses to your frequently asked questions with input from Genentech/Roche. This FAQ document can be accessed on our website at the following link: https://hdsa.org/wp-content/uploads/2018/10/FINAL-101518_FAQ-about-Upcoming-Roche-Studies_for-HDSA.pdf Thanks again…

ASOs therapies have dominated the news recently, but did you know that there are many additional strategies being developed in HD labs for huntingtin lowering? PhD student Julia Alterman will give a webinar next week about a method for interfering with huntingtin RNA and how she is testing it in animal models of HD. Julia…

When symptoms of HD start before the age of 20, this is known as juvenile onset Huntington’s disease, or JHD. A team in Italy led by Dr. Fernando Squitieri did a large analysis of data from big studies like REGISTRY and ENROLL, comparing JHD with adult HD and revealing new insights into how Huntington’s affects…

If you missed the clinical trials update webinar presented by Roche on Wednesday the 26th, it’s now available for viewing on HDSA’s youtube channel and will be uploaded to our website shortly. In this webinar, four Roche team members, J.P. Sacksteder, Mai-Lise Nguyen, Erik Lundgren, and Dr. Scott Schobel, provided a clear community update on…

This past Sunday at the plenary meeting of the European Huntington’s Disease Network (EHDN), Roche Pharmaceuticals announced some details about two upcoming clinical trials. This scientific presentation in Vienna was followed by a Community Statement from Roche/Genentech to HD families worldwide that HDSA shared via our website, social media, and email. In summary, Roche will…

This week, members of the HDSA team are attending the 2018 plenary meeting of the European Huntington’s Disease Network (EHDN) in Vienna, Austria. Alongside the latest findings from HD researchers worldwide, Ionis Pharmaceuticals and Roche/Genentech will continue to present the results from the successful safety trial of their huntingtin-lowering ASO. The HD research community is…

This week, meet Dr. Rachel Harding from the University of Toronto, the recipient of this year’s Berman-Topper Family HD Career Development Fellowship. In our latest Researcher Spotlight, we asked Dr. Harding about her work, her goals, and her life outside the lab. She believes strongly in openly sharing scientific data, and you can also follow…

The prestigious Science magazine published an article on Huntington’s disease this week, describing the huntingtin-lowering ASO drug developed by Ionis, and profiling the very first trial participant. The article covered her family history with HD and her current experience in the open-label trial, and featured discussions with her physician Dr. Blair Leavitt, a researcher at…

This week, the FDA released a proposal to streamline the way they oversee the evaluation of protocols related to gene therapy. Essentially the US government has recognized that gene therapies are an important emerging class of medicines, and they’re trying to re-vamp the administrative process so that it’s shorter and less repetitive. Several NIH and…

This week marked the first FDA approval of a gene silencing drug, for a rare hereditary disease called ATTR amyloidosis. A successful trial determined that the drug, ONPATTRO (patisiran), helped with people’s symptoms, like polyneuropathy (pain from nerve damage), dizziness, and stomach issues, and improved quality of life, activities of daily living, and nutritional status….