News

Posting from uniQure press releases: April 17, 2025 ~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory…

Make sure to read these articles related to Huntington's disease if you want to learn more! "A Faustian bargain" in the Economist: https://www.economist.com/science-and-technology/2015/03/05/a-faustian-bargain "Is Huntington’s Disease an Unintended Consequence of…

Sarah Hernandez, PhD, at the Hereditary Disease Foundation, discusses clinical trials and recent research for Huntington's disease. A fantastic culmination from hard working scientists around the world. Watch below. https://www.youtube.com/watch?v=wALTraiLmNg

Data from HD-YAS,  a deep-phenotyped longitudinal study of young stages 0 and 1 Huntington's disease gene expanded (HDGE) individuals, helps to identify predicted clinical motor diagnosis, and biomarkers of disease…

Scientists at the Broad Institute of MIT and Harvard, Harvard Medical School, and McLean Hospital have discovered a mechanism by which the inherited genetic mutation in Huntington's disease leads to…

Congrats to the Thompson Lab on their recent discovery published in Nature Neuroscience discovering molecular mechanisms driving RNA processing defects in Huntington's disease! Read here: https://www.nature.com/articles/s41593-024-01850-w?utm_source=rct_congratemailt&utm_medium=email&utm_campaign=oa_20250106&utm_content=10.1038/s41593-024-01850-w

HD-CARE and HDSA OC are cohosting a fantastic community lecture on February 19th with Dr. Sarah Tabrizi from University of College London. Register today: https://shorturl.at/Yj8uZ

Read full release here: https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-enters-global-license-and-collaboration

Never too late to post good news! Dr. Leslie Thompson along with Drs. Beverly Davidson and Jang-Ho Cha received a 2023 $1M Transformative Research Award taking strides towards treatments for…