Articles with the topic: featured

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

Huntington’s disease therapeutics conference 2022 - Day 3

Huntington’s disease therapeutics conference 2022 - Day 3

Check out research updates from Day 3 of the 2022 HD Therapeutics Conference #HDTC2022

Dr Sarah Hernandez, Dr Leora Fox, Dr Rachel Harding, and Joel StantonMarch 08, 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

KINECT-HD trial shows valbenazine improves involuntary movements in Huntington's disease

KINECT-HD trial shows valbenazine improves involuntary movements in Huntington's disease

In a much-needed bit of good news for the Huntington's disease community, Neurocrine Bioscience's KINECT-HD trial showed that treatment with valbenazine significantly reduced the involuntary movements called chorea

Professor Ed WildDecember 14, 2021

“Seeing” the toxic huntingtin protein in people with HD

“Seeing” the toxic huntingtin protein in people with HD

New tools let us “see” clumps of toxic huntingtin protein which build up in the brains of people with Huntington’s disease over time. Tracking these clumps might help us to better understand how HD progresses or how treatments might slow or halt HD.

Dr Rachel HardingDecember 08, 2021

Real talk: Q&A with Roche about GENERATION-HD1

Real talk: Q&A with Roche about GENERATION-HD1

The HDBuzz team sat down (virtually) for an in depth Q&A session with the team at Roche to answer questions about tominersen and the recent halting of the GENERATION-HD1 trial

Dr Sarah Hernandez and Dr Leora FoxSeptember 28, 2021

Another tool in the box: Creation of a molecular “dimmer switch” advances gene editing

Another tool in the box: Creation of a molecular “dimmer switch” advances gene editing

A new system has been developed that allows researchers to fine-tune gene expression with oral drugs, work that provides a powerful tool for gene editing.

Dr Leora FoxAugust 30, 2021