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Australia Opens The Door for SKY-0515: Skyhawk Seeks Provisional Approval for Its Oral HD Drug
Latest News
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The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy
⏱️ 10 min read | The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required.
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A new way of thinking about trials to prevent Huntington's disease
Can we test drugs to delay or prevent the onset of Huntington's disease? New research suggests it's possible
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New roles for huntingtin: removing a healthy protein to understand its function
Completely removing normal huntingtin in adults may disrupt healthy brain function, a recent study suggests.
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Precision huntingtin-lowering drug trials target the mutant protein
WAVE Life Sciences launches PRECISION clinical trial to suppress the mutant Huntington's disease protein
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A step forward for gene editing: CRISPR-Cas9 and HD
Evolving CRISPR-Cas9 techniques can now be used to edit the HD gene in a living mouse brain.
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Update confirms Huntington's disease 'gene silencing' trial on track
Ionis says its trial of HTTRx, intended to lower huntingtin protein, is fully recruited and plans to extend it
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New study reveals a potential HD biomarker
A potential HD biomarker has been uncovered in a recent clinical study
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Has a "wonder drug" for dementia been discovered? (Spoiler alert: no.)
Media reports of a wonder drug for neurodegenerative diseases like Huntington's disease are overhyped
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Huntington's Disease Therapeutics Conference 2017 – Day 3
HDBuzz summarises final day of the 2017 Huntington's Disease Therapeutics Conference in Malta
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Huntington's Disease Therapeutics Conference 2017 – Day 2
HDBuzz summarises all the science from the 2017 Huntington's Disease Therapeutics Conference in Malta – day 2
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Huntington's Disease Therapeutics Conference 2017 – Day 1
HDBuzz summarises all the science from the 2017 Huntington's Disease Therapeutics Conference in Malta – day 1
HDBuzz Trial Tracker
Progress update for NCT06585449 – A Study to Evaluate ALN-HTT02
Additional insights from Phase 2 study results and update on plans for Phase 3 trial for votoplam
