Check out Dancing at the Vatican! An inspirational documentary telling the story of a seemingly impossible journey made by families bound by the same devastating, genetically inherited disease, Huntington's. Leaving…
uniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington’s Disease
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…
Want to learn more about HD?
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…
HDF Spotlight Webinar: Launching Hope from the Lab, HD Research Breakthroughs with Dr. Sarah Hernandez
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
New HD CSF biomarkers informing clinical trials from Dr. Tabrizi Lab
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…
“A new understanding of Huntington’s disease” – Research from Broad Institute
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…
New research from the Thompson Lab!
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
Save the Date! Dr. Sarah Tabrizi Community Lecture on February 19
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
PTC Therapeutics Enters into a Global License and $1B Collaboration Agreement with Novartis for PTC518 Huntington’s Disease Program
Read full release here: https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-enters-global-license-and-collaboration
Leslie Thompson Speaks at 2024 HDF Gala
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…