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….

On August 4th, Roche Pharmaceuticals announced that their huntingtin-lowering therapy was granted PRIME status from the European Medicines Agency (EMA). The EMA is the European equivalent of the United States Food and Drug Administration (FDA). In Europe, PRIME (PRIority MEdicines) designation means that the EMA has agreed that this drug, RG6042 (formerly IONIS-HTTRx) could potentially…

Community research surveys are valuable ways for researchers to get input from HD patients, families and caregivers about a variety of topics, and can help guide strategies for HD research and care. HDSA works with scientists and students to make their university-approved research surveys available for easy online participation through our website. This week, there’s…

At-home DNA kits like 23 & Me are growing in popularity, and claim to provide information about your health and ancestry. The Washington Post created an FAQ about what you can actually expect to learn – and who gets your info. These kits do not test for genetic disorders like Huntington’s disease. For more information…

The Rare Disease Report covers stories about medicine, finance, and advocacy in rare disease fields. Last week an article explored some of the promising therapies in the pipeline for HD. To learn more about advances in HD research, check out HDSA’s page detailing current preclinical and clinical research studies and their current phase of development….