Articles with the topic: huntingtin-lowering

uniQure gets the green light to resume testing HD gene therapy

uniQure gets the green light to resume testing HD gene therapy

Following a 3-month pause in enrollment due to concerns about side effects, uniQure shared the good news that their trial of the HD gene therapy AMT-130 will continue as planned, with new safety measures in place.

Dr Leora FoxNovember 03, 2022

Update on the PTC Therapeutics PIVOT-HD Trial

Update on the PTC Therapeutics PIVOT-HD Trial

Last week, PTC Therapeutics released a statement sharing that recruitment of participants into the US arm of the PIVOT-HD trial has been paused. In this article, we will lay out exactly what is known and what this announcement means.

Dr Rachel Harding and Dr Leora FoxNovember 03, 2022

Forward momentum for Roche and Wave in latest news about huntingtin-lowering trials

Forward momentum for Roche and Wave in latest news about huntingtin-lowering trials

Two companies developing medicines for Huntington’s disease announced news about their huntingtin-lowering drugs. Let’s talk about the history and the caveats surrounding these hopeful pieces of news.

Dr Jeff Carroll and Dr Leora FoxSeptember 30, 2022

Hereditary Disease Foundation (HDF) conference 2022 – Day 1

Hereditary Disease Foundation (HDF) conference 2022 – Day 1

Read updates from clinical trials and scientific research on Huntington’s disease from Day 1 of the 2022 HDF Milton Wexler Biennial Symposium #HDF2022

Dr Rachel Harding and Dr Sarah HernandezAugust 31, 2022

Sad news from Novartis: dosing suspended in VIBRANT-HD trial of branaplam

Sad news from Novartis: dosing suspended in VIBRANT-HD trial of branaplam

HDBuzz is saddened to share the news that the VIBRANT-HD trial of the oral huntingtin-lowering drug, branaplam, has been temporarily suspended for the safety of participants.

Dr Leora FoxAugust 08, 2022

A spoonful of branaplam helps the huntingtin go down

A spoonful of branaplam helps the huntingtin go down

Branaplam was originally designed to treat spinal muscular atrophy, but a new paper outlines how it could hold promise for treating Huntington’s. This oral drug lowers huntingtin protein and will now be tested in a study called VIBRANT-HD.

Dr Rachel HardingMarch 16, 2022

Shining a spotlight on huntingtin: a tool to measure huntingtin-lowering in real time

Shining a spotlight on huntingtin: a tool to measure huntingtin-lowering in real time

A new imaging tool means that scientists can now directly measure the levels of the toxic huntingtin protein in animal models of Huntington’s disease, letting us see how well huntingtin lowering therapies are working in their brains

Dr Rachel HardingFebruary 17, 2022

Oral drug may change the story for huntingtin lowering

Oral drug may change the story for huntingtin lowering

Researchers with PTC Therapeutics recently published exciting new findings - a promising new huntingtin lowering drug that can be taken as a pill. Will this change how we move forward with huntingtin lowering?

Dr Sarah Hernandez and Dr Jeff CarrollFebruary 01, 2022

Finding the silver lining: an update on the Roche GENERATION-HD1 trial data

Finding the silver lining: an update on the Roche GENERATION-HD1 trial data

The first round of findings from the halted tominersen huntingtin lowering trial, GENERATION-HD1, run by Roche were shared this week with the HD community. HDBuzz explains what they found and what’s next.

Dr Leora Fox and Dr Rachel HardingJanuary 21, 2022