Articles with the topic: drug-development

Huntington's Disease Therapeutics Conference 2024 - Day 1

Huntington's Disease Therapeutics Conference 2024 - Day 1

Check out research updates from Day 1 of the 2024 HD Therapeutics Conference #HDTC2024

Dr Leora Fox, Dr Rachel Harding, and Dr Sarah HernandezMarch 05, 2024

CRISPR-based drugs: one giant leap for mankind

CRISPR-based drugs: one giant leap for mankind

Casgevy is the first CRISPR-based drug to make its way through the approval process, all but curing Sickle Cell Disease and it’s paving the way for similar drugs targeting other diseases. Is Huntington’s disease next?

Dr Sarah HernandezFebruary 14, 2024

Putting it in print: GENERATION HD1 study results published

Putting it in print: GENERATION HD1 study results published

Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and what’s next?

Dr Leora Fox, Dr Rachel Harding, and Dr Sarah HernandezDecember 07, 2023

Beyond huntingtin lowering: out-of-the-box approaches for the treatment of HD

Beyond huntingtin lowering: out-of-the-box approaches for the treatment of HD

The HD pipeline is rich and varied. Let's talk about some out-of-the-box approaches for developing drugs for HD that don't involve huntingtin lowering.

Kelly AndrewNovember 24, 2023

Drug to treat movement symptoms of HD approved by FDA

Drug to treat movement symptoms of HD approved by FDA

The FDA has approved valbenazine, also known as INGREZZA, as a treatment for the movement symptoms of Huntington’s disease

Dr Leora Fox and Dr Rachel HardingAugust 22, 2023

Disappointing news from Novartis about branaplam and the VIBRANT-HD trial

Disappointing news from Novartis about branaplam and the VIBRANT-HD trial

Novartis have announced that they are ending development of the drug branaplam in Huntington’s disease. Here, we review this latest news and its impact on the HD community.

Dr Rachel HardingDecember 09, 2022

Artificial intelligence to save the day? How clever computers are helping us understand Huntington’s disease.

Artificial intelligence to save the day? How clever computers are helping us understand Huntington’s disease.

Scientists at IBM and the CHDI Foundation have used artificial intelligence to analyse datasets from Huntington’s disease observational trials to model progression of the disease. They hope their findings will help improve clinical trial design.

Dr Rachel HardingJune 21, 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

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

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