Scottish Huntington’s Association hails announcement of breakthrough Huntington’s disease treatment
Research published by drug company uniQure today (24 September 2025) shows that its treatment AMT130 is safe, well tolerated, and slows progression of Huntington’s disease by around 75%.
Commenting on the news CEO of Scottish Huntington’s Association, Alistair Haw, said:
“These are the best results to date from any clinical trial aiming to slow the progression of Huntington’s disease. After many years of hope and false dawns, this is a day for rejoicing for the global Huntington’s community. The first treatment for this horrendous condition appears to finally be appearing at the end of this long dark tunnel.”
However, Mr Haw warned of the need to ensure that Scotland’s health and social care system is ready for the treatment:
“At present, people in a Huntington’s family who know they are at risk of inheriting the disease from a parent overwhelmingly choose not to be tested to find out if they have the faulty gene. With a treatment on the horizon, this will all change. More people will come forward requiring specialist help and support with this process. This will require more capacity in the system to cope with the demand, not less.
“Sadly, Glasgow City HSCP is planning to become the first area in history to defund our Huntington’s Disease Specialist Service. This was always an ill-advised false economy, but in light of today’s news this plan becomes an ill-advised false economy on steroids.
“After decades of progress Scotland is a world leader in providing specialist Huntington’s care and support thanks to services provided by Scottish Huntington’s Association backed by a Scottish Government supported National Care Framework. At this time of all times we need to grow these services, not reduce them. Unless health and social care providers grasp this reality, and now, Scotland risks snatching defeat from the jaws of victory on this otherwise unprecedented day of celebration for the Huntington’s community. And that would be unforgiveable. We call on the Scottish Government to unite with us to prevent this from happening.”
About today’s announcement from UniQure – Q&A
What has been announced?
On Wednesday 24 September 2025, in an announcement of the results of three years’ treatment in the trial, we learned that AMT130 was safe and well tolerated, and slows progression of Huntington’s disease by around 75% as measured using the composite unified HD rating scale.
Treated patients also had biomarker changes suggesting the effect of the drug comes from its ability to rescue neurons. Specifically, the level of neurofilament (a marker of neuronal damage) was below baseline at the 3 year time point, indicating that the rate of damage was slower at the end of the trial than at the beginning, as a result of treatment.
What is AMT130?
AMT130 is an experimental gene therapy, developed by the US company UniQure, that reduces production of mutant huntingtin, the harmful protein that causes Huntington’s disease (HD). AMT130 is administered as a single dose via injection directly into the brain, in a neurosurgical operation lasting 12+ hours. It has been under test in a small clinical trial since 2019.
What does this announcement mean?
These are the best results we’ve seen from any clinical trial aiming to slow the progression of Huntington’s disease. It is the view of the world renowned Huntington’s Disease Centre of University College London that the data convincingly suggest that AMT130 is truly altering the progression of Huntington’s disease. This is a fabulous result for the whole Huntington’s community.
We do not yet know whether AMT130 will delay onset in people with the Huntington’s genetic mutation that causes Huntington’s disease but who don’t yet have symptoms. It should – but this has not yet been tested.
What happens next?
UniQure intends to rapidly apply for regulatory approval for AMT130 so that it can be prescribed by doctors. But it will take time to turn this success into a treatment that is widely available.
While nothing is set in stone, UniQure intends to apply for approval immediately in the USA and, if successful, approval there would likely happen in 2026. We expect UniQure to apply for approval in the EU and UK as well. That will likely take longer, and we have no information about the timeline yet.
The UK’s authority for approving medicines for safety, quality, and effectiveness is the Medicines and Healthcare products Regulatory Authority (MHRA). If it approves AMT130 for prescribing in the UK, we will only be able to use it on the NHS in Scotland if the Scottish Medicines Consortium (SMC) also approves it after a cost versus effectiveness analysis. [The equivalent approval body for the rest of the UK is the National Institute for Health and Care Excellence (NICE)] This process could take several years.
We will be pushing hard at every level to get the treatment as soon as possible to all people in Scotland who could benefit. But until MHRA and SMC approve AMT130, it cannot be prescribed and we cannot enter into any discussion about how to access it.
How can we find out more?
Further public updates will be given as more information emerges. UniQure’s patient liaison team can be contacted at medinfo@uniqure.com
We would also recommend visiting https://en.hdbuzz.net/ for further updates.
*Scottish Huntington’s Association thanks Prof Sarah Tabrizi and Prof Ed Wild of the Huntington’s Disease Centre at University College London for their guidance in compiling this information.*