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

An Updated Trial Design for Roche's Huntingtin Lowering Trial

An Updated Trial Design for Roche's Huntingtin Lowering Trial

Roche announces an update to the GENERATION HD1 trial after reviewing the ASO open label extension study

Dr Jeff CarrollMarch 21, 2019

When interrupting is good: genetic hiccups that protect against Huntington's disease

When interrupting is good: genetic hiccups that protect against Huntington's disease

Multiple teams find small differences in the 'CAG repeat' bit of the Huntington's disease gene. They don't directly change the huntingtin protein, but do alter the age of symptom onset. What's behind this enigma and what does it mean for patients?

Dr Jeff CarrollMarch 14, 2019

Huntington's disease therapeutics conference 2019 - Day 3

Huntington's disease therapeutics conference 2019 - Day 3

New insights into the huntingtin protein, and further progress in turning genetic quirks that modify the course of HD into treatments, on the final day of the therapeutics conference

Joel Stanton and Dr Jeff CarrollMarch 05, 2019

Huntington's disease therapeutics conference 2019 - Day 2

Huntington's disease therapeutics conference 2019 - Day 2

New tools to bridge the gap between the lab and patients in our update from day 2 of the 2019 HD Therapeutics conference

Joel Stanton and Dr Jeff CarrollFebruary 28, 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

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