clinical-trial
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Putting Huntingtonās disease medications to the test: results from the Neuro-HD trial
ā±ļø6 min read | A clinical trial, Neuro-HD, compared 3 common drugs for HD symptoms. The 1 year study found no ābestā medication, but clear differences between treatments. The results support a personalised, symptom-by-symptom approach to HD treatment.
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First participants dosed in new POINT-HD huntingtin-lowering trial
POINT-HD has begun dosing its first participants with the drug RG6496, marking an early but important step for a new selective huntingtin-lowering approach.
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SOM3355 Moves Toward Phase 3 as Both EMA and FDA Signal Support
SOM3355, a drug to help manage HD symptoms, has received a positive opinion from the EMA for orphan drug designation, and the company is aligned with the FDA after its End-of-Phase-2 meeting. A global Phase 3 trial is expected to start in 2026.
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Tooth fairy therapy? Stem cells from teeth tested in a small Huntingtonās disease trial
A trial tested stem cells from human teeth as a possible treatment for HD. The approach appeared safe with some improvements in symptoms, but the small size of the study and the unclear underlying science of this approach means caution is urged.
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UniQure and FDA No Longer in Alignment on Approval Pathway for AMT-130
In a new press release, uniQure shared that the FDA no longer agrees with their approach for advancing AMT-130, specifically the use of an external control group. The path forward is uncertain, but uniQure remains committed to the HD community.
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Additional Clarity: What We Know 4 Weeks After the uniQure News
Weāre 4 weeks out from uniQureās positive update about AMT-130 that took the world by storm. Does the data match the media hype? Where do uncertainties remain? And what are some of the key questions from the Huntingtonās disease community?
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Huntingtonās Disease Clinical Research Congress 2025 ā Day 3
Catch up on all the latest HD news from Day 3 of the Huntingtonās Disease Clinical Research Congress in Nashville, USA.
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Huntingtonās Disease Clinical Research Congress 2025 ā Day 2
Catch up on all the latest HD news from Day 2 of the Huntingtonās Disease Clinical Research Congress in Nashville, USA.
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Huntingtonās Disease Clinical Research Congress 2025 – Day 1
Catch up on all the latest HD news from Day 1 of the Huntingtonās Disease Clinical Research Congress in Nashville, USA.
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The First Domino Falls: AMT-130 Gene Therapy Slows Huntingtonās in Landmark Trial
In an update from uniQure, they report that their experimental gene therapy, AMT-130, has the potential to slow Huntington’s disease progression in key clinical study.