drug-development
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Putting it in print: a clinical trial trying to rein in runaway pruning
9 min read
Four years after announcing positive results in their trial of ANX005 (tanruprubart) for HD, Annexon has formally published the results. It’s safe, hits its target, and may have helped some participants – so what’s the holdup?
By Dr Leora Fox -
The Other Shoe Drops: uniQure Shares Plans To Submit Licensing Application With The FDA for AMT-130
8 min read
After a turbulent past year, uniQure have shared encouraging news that the FDA has agreed their Phase I/II study data can serve as the basis for an application that could lead to the approval of AMT-130 in the US.
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Prilenia’s Application for European Approval of Pridopidine Withdrawn
4 min read
Prilenia’s application for European approval of pridopidine was quietly withdrawn 6 months ago, in November 2025, after the EMA had already recommended refusing the drug. Here’s what happened, and why you’re only hearing about it now.
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A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK
5 min read
While the US regulatory path for AMT-130 remains complicated, uniQure announced plans to seek approval for its HD gene therapy in the UK. Here’s what that means, and what might be to come.
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Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam
9 min read
PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to 52% slowing of disease progression. Here’s what the data show, what’s still missing, and the roll out of the Phase 3 trial.
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Australia Opens The Door for SKY-0515: Skyhawk Seeks Provisional Approval for Its Oral HD Drug
7 min read
An oral Huntington’s disease drug is eligible for accelerated approval in Australia. This isn’t full approval yet, but it opens a faster path to potentially getting this once-daily pill to people with HD sooner.
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UniQure Receives FDA Meeting Minutes on AMT-130 While Community Support Remains Strong
5 min read
UniQure received FDA’s meeting minutes on AMT-130. While there were no new updates, the community’s response has been powerful, with 41K+ petition signatures & unified advocacy from major HD orgs.
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UniQure and FDA No Longer in Alignment on Approval Pathway for AMT-130
8 min read
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
19 min read
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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Controlling DNA Scanning Machines to Slow Expansion of CAG Repeats
5 min read
A new study identifies small molecules that target a DNA repair machine, opening a potential path to delay Huntington’s disease onset.