Articles with the topic: featured

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

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 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)

Dr Leora FoxOctober 19, 2018

Success! ASO drug reduces levels of mutant protein in Huntington's disease patients

Success! ASO drug reduces levels of mutant protein in Huntington's disease patients

Amazing news from Ionis and Roche! HTTRx drug successfully lowers harmful huntingtin protein in spinal fluid

Dr Jeff CarrollDecember 11, 2017

Switch off the genome editor when you’re done

Switch off the genome editor when you’re done

CRISPR genome editing just got an off-switch. We cut through the hype to explore the technology in HD

Dr Michael FlowerNovember 02, 2017

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