drug-development
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Huntington's Disease Therapeutics Conference 2024 – Day 1
Check out research updates from Day 1 of the 2024 HD Therapeutics Conference #HDTC2024
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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?
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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?
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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.
By Kelly Andrew -
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
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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.
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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.
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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.
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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?
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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.