Huntington's disease goes viral as UniQure inches ahead in gene therapy race

Huntington's disease goes viral as UniQure inches ahead in gene therapy race

FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients

Dr Jeff CarrollJanuary 30, 2019

Advances on many fronts in the battle against the protein that causes Huntington's disease

Advances on many fronts in the battle against the protein that causes Huntington's disease

This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager

Dr Jeff CarrollDecember 04, 2018

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)

Dr 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

Disappointing news from LEGATO-HD trial of laquinimod in Huntington's disease

Disappointing news from LEGATO-HD trial of laquinimod in Huntington's disease

The LEGATO-HD trial of laquinimod did not slow progression of Huntington's disease. Here's the lowdown.

Dr Jeff CarrollAugust 02, 2018

HDBuzz Enrolls in Enroll-HD

HDBuzz Enrolls in Enroll-HD

As promised, HDBuzz's Jeff Carroll finally signed up for the Enroll-HD study. Learn more about this important global HD "observational study" here.

Dr Jeff CarrollJuly 31, 2018

First Dolly, now Piglet; a new Huntington’s disease pig knock-in model confirmed

First Dolly, now Piglet; a new Huntington’s disease pig knock-in model confirmed

For the first time in HD research, an animal model has been created in pigs using 2 cutting edge DNA editing t

Caroline CaseyJuly 10, 2018

New collaboration seeks to speed Huntington's disease drug licensing

New collaboration seeks to speed Huntington's disease drug licensing

Critical Path Institute launches new initiative to get HD drugs licensed as quickly as possible

Dr Jeff CarrollMay 10, 2018

HDSA’s Q&A on the latest huntingtin-lowering update from Ionis and Roche

HDSA’s Q&A on the latest huntingtin-lowering update from Ionis and Roche

Our friends at @hdsa have produced this really good Q&A document about the latest huntingtin-lowering news.

Dr Leora FoxApril 26, 2018

Exciting Experiments in "Open Science" by Huntington's Researchers

Exciting Experiments in "Open Science" by Huntington's Researchers

Brave scientists open their notebooks to help drive HD research at the risk of getting "scooped".

Anna Marie YannyApril 20, 2018

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