clinical-trial
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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 Huntington’s 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
We’re 4 weeks out from uniQure’s 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 Huntington’s disease community?
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Huntington’s Disease Clinical Research Congress 2025 – Day 3
Catch up on all the latest HD news from Day 3 of the Huntington’s Disease Clinical Research Congress in Nashville, USA.
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Huntington’s Disease Clinical Research Congress 2025 – Day 2
Catch up on all the latest HD news from Day 2 of the Huntington’s Disease Clinical Research Congress in Nashville, USA.
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Huntington’s Disease Clinical Research Congress 2025 – Day 1
Catch up on all the latest HD news from Day 1 of the Huntington’s Disease Clinical Research Congress in Nashville, USA.
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The First Domino Falls: AMT-130 Gene Therapy Slows Huntington’s 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.
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SKY-0515 Lowers Huntingtin In People With Huntington’s Disease In Trial Update
SKY-0515, an oral drug, safely lowers huntingtin in people with HD and may also reduce PMS1. This could offer a possible two-pronged approach to treat HD. SKY-0515 is now being tested in a larger Phase 2/3 trial.
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PROOF in the Publishing: Slicing the Data on Pridopidine
Pridopidine has been tested as a potential HD treatment over decades. The large PROOF-HD trial did not meet its main goals, but its results are now published. Publication makes the full data available, helping the HD community learn from the study.
By Dr Leora Fox