Articles with the topic: featured

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

Unpacking recent gene therapy press

Unpacking recent gene therapy press

Voyager Therapeutics is shifting towards a new technology to deliver gene therapy and away from a planned HD clinical trial. But this could lead to less invasive drugs in the long run, and many other companies are working on HD gene therapies.

Dr Leora Fox and Dr Sarah HernandezAugust 16, 2021

Does blood hold the key to testing treatments earlier in HD patients?

Does blood hold the key to testing treatments earlier in HD patients?

A new study by researchers at Johns Hopkins describes a non-invasive way to track progression of Huntington’s disease. This could be used before patients even start showing symptoms to help test treatments in early stages of disease.

Dr Rachel HardingAugust 04, 2021

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

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