Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine
Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.
UniQure announces key details of its planned trial to assess the safety and ability of AMT-130 gene therapy to lower the problematic huntingtin protein using a ‘single-shot’ virus delivery system.
Hot off the presses - New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO
HDBuzz reports from the annual Huntington’s disease therapeutics conference in Palm Springs
HDSA's frequently asked questions factsheet about the huntingtin-lowering trial ASO program was so good we stole it (with permission)
Finally, a big study that shows what childhood HD looks like. This will help us work out if new drugs work in children too
The GENERATION-HD1 trial will test whether RG6042 – formerly Ionis-HTTRx – slows the progression of Huntington's disease