Read updates from clinical trials and scientific research on Huntington’s disease from Day 1 of the 2022 HDF Milton Wexler Biennial Symposium #HDF2022
After receiving a high dose of uniQure’s gene therapy for Huntington’s disease, a few patients experienced serious side effects, but are now recovering. HDBuzz explores what this means for the experimental huntingtin-lowering drug, AMT-130.
Disappointing news from Roche and Ionis; the phase III Tominersen huntingtin-lowering trial has been halted early
FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
Media reports of a wonder drug for neurodegenerative diseases like Huntington's disease are overhyped
Work with genome editing techniques (zinc fingers and CRISPR) brings these tools closer to use in HD clinical trials
Major Announcement: First HD patients treated with gene silencing drugs
How will we know whether HD-lowering treatments are working? New advancements in the detection of mutant huntingtin.