Articles with the topic: featured

A first for CRISPR gene editing could have wider applications for human disease

A first for CRISPR gene editing could have wider applications for human disease

A successful early trial of a drug for Familial Transthyretic Amyloidosis showed that CRISPR gene editing could be safely used in the human body. What does this mean for gene editing in HD?

Daniel O’ReillyJuly 21, 2021

Scientists identify precisely how pridopidine works in models of Huntington’s disease

Scientists identify precisely how pridopidine works in models of Huntington’s disease

New studies pinpoint precisely how the drug pridopidine works in models of Huntington’s disease

Dr Rachel HardingJune 12, 2021

Huntington's disease therapeutics conference 2021 - Day 3

Huntington's disease therapeutics conference 2021 - Day 3

Catch up on all of the latest updates from day 3 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021

Dr Rachel Harding, Dr Leora Fox, and Professor Ed WildApril 29, 2021

Huntington’s disease therapeutics conference 2021 - Day 1

Huntington’s disease therapeutics conference 2021 - Day 1

Catch up on all of the latest updates from day 1 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021 including presentations from Wave, Roche and Triplet.

Dr Rachel Harding, Dr Leora Fox, and Dr Jeff CarrollApril 28, 2021

Huntington's disease therapeutics conference 2021 - Day 2

Huntington's disease therapeutics conference 2021 - Day 2

Catch up on all of the latest updates from day 2 of the 2021 CHDI Huntington’s disease therapeutics conference #HDTC2021

Dr Rachel Harding, Dr Leora Fox, Dr Jeff Carroll, and Professor Ed WildApril 28, 2021

Huntington’s disease clinical trial round up

Huntington’s disease clinical trial round up

Catch up on all the latest Huntington's disease clinical trial news in this one stop shop article covering all of the recent developments in making medicines for Huntington's disease

Dr Rachel HardingApril 26, 2021

Disappointing Results from Wave’s PRECISION-HD1 and 2 Trials

Disappointing Results from Wave’s PRECISION-HD1 and 2 Trials

Wave Life Sciences shared the disappointing news that their two ASOs in Phase 1/2 trials in HD patients did not successfully lower mutant huntingtin.

Dr Rachel Harding and Dr Leora FoxMarch 30, 2021

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