Articles with the topic: huntingtin-lowering

Exciting new Huntingtin lowering tool described

Exciting new Huntingtin lowering tool described

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.

Dr Jeff CarrollAugust 05, 2019

Details emerge of first Huntington’s disease gene therapy clinical trial

Details emerge of first Huntington’s disease gene therapy clinical trial

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.

Dr Anna PfalzerJuly 17, 2019

Fresh Updates from First Huntingtin Lowering Study Publication

Fresh Updates from First Huntingtin Lowering Study Publication

Hot off the presses - New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO

Dr Jeff CarrollMay 07, 2019

Huntington's disease therapeutics conference 2019 - Day 1

Huntington's disease therapeutics conference 2019 - Day 1

HDBuzz reports from the annual Huntington’s disease therapeutics conference in Palm Springs

Joel StantonFebruary 27, 2019

HDSA FAQ on the Roche/Genentech RG6042 program

HDSA FAQ on the Roche/Genentech RG6042 program

HDSA's frequently asked questions factsheet about the huntingtin-lowering trial ASO program was so good we stole it (with permission)

Leora FoxOctober 19, 2018

Kids sometimes get Huntington’s disease too

Kids sometimes get Huntington’s disease too

Finally, a big study that shows what childhood HD looks like. This will help us work out if new drugs work in children too

Dr Michael FlowerOctober 10, 2018

Roche announces details of its 'pivotal' huntingtin-lowering study

Roche announces details of its 'pivotal' huntingtin-lowering study

The GENERATION-HD1 trial will test whether RG6042 – formerly Ionis-HTTRx – slows the progression of Huntington's disease

Dr Jeff CarrollSeptember 17, 2018