We all have days when we feel irritable, like when you’re stuck in traffic, or someone cuts in line at the grocery store. But for people living with Huntington’s disease (HD), irritability isn’t just a passing mood. It can feel like a thunderstorm that arrives suddenly, often without warning, and affects not only the person with HD but also everyone around them.

In a recent study, Dr Sarah Gunn and her team of researchers from Leicester, UK, set out to explore what irritability really feels like for people with HD and their families. By hearing directly from people with HD, the researchers painted a clearer, more honest picture of irritability for folks impacted by this disease. This study showed that irritability is much more than just a mood. It is a real and challenging problem that impacts emotional regulation, relationships, and general wellbeing.

It’s Like a Volcano 

One common, but lesser-understood symptom of HD is irritability. Irritability can be deeply distressing for the person with HD, who is experiencing these intense emotions. However, it can also cause significant emotional strain for family and friends, leaving everyone involved feeling frustrated, isolated, and misunderstood.

Imagine holding a bottle of fizzy drink (soda) that’s been shaken. You try to keep it closed, but eventually, the pressure builds, and it explodes. This image helps us understand how irritability feels for people with HD: sudden, intense, and difficult to control. It’s like a volcano bubbling beneath the surface, calm one moment, then suddenly erupting over something that might seem small or insignificant to others. It’s not that people with HD want to lash out; they may feel helpless because they notice their irritation rising but cannot control their reactions.

Inside the Storm

Instead of relying on medical tests or numbers, Dr Gunn and her team wanted to understand irritability from the perspective of those who live with HD. They investigated irritability in people with pre-manifest and manifest HD. ‘Pre-manifest’ HD refers to people who have tested positive for the gene that causes HD (CAG number is 40 or more), but they do not yet display any recognisable symptoms (equivalent to HD Integrated Staging System (HD-ISS) Stage 0). Whereas ‘manifest’ HD refers to people who have tested positive for the HD gene and the person is showing recognisable symptoms of HD (HD-ISS Stage 1 and beyond). In this study, 42% of individuals had pre-manifest HD, while 58% had manifest HD.

The researchers conducted interviews, giving people with HD a chance to share their experiences, in their own words. The conversations covered how people with HD felt about the word “irritability,” how they experienced it, what triggered it, how they managed it, and how it affected their relationships.

Once the interviews were complete, the researchers carefully reviewed the interviews, focussing on irritability and how it affected people with HD. They identified three main themes (categories) that came up repeatedly:

1. The Triggers
2. The Challenges
3. Soothing the Struggle 

The Triggers 

Dr Gunn and her team of researchers noted common ideas across different participants with HD, for the category, ‘The Triggers’. One key idea was, ‘It’s not me, it’s the HD’. Participants had complicated feelings about the word “irritability.” Some participants felt uncomfortable identifying with irritability, as it can carry negative connotations, while others felt it accurately described their experience with HD.

Many participants explained how the stress of living with HD made them feel more irritable. Worries about their future and concerns for their family members added to the strain, making it harder to stay calm, and increasing the chance of snapping at others.

Feeling out of control or overwhelmed could contribute to heightened feelings of irritability in people with HD. Participants commented that life events such as grief, past trauma, or busy, chaotic environments, made it harder to manage emotions. Interestingly, all participants talked about how basic physical needs, like being hungry, tired, in pain, or too hot or cold, could quickly push their irritability over the edge. It’s a bit like how anyone might feel more impatient or upset when they are hungry, or in pain.

The Challenges

Participants remarked how even tiny annoyances, like a door slamming while you are trying to talk, could quickly explode into intense anger. These feelings might linger, making it hard to stay calm or think clearly. This could lead to snapping or saying hurtful things. Some felt powerless over these feelings, which left them scared and worried about their own outbursts and how it affected those around them.

Irritability in HD can cause misunderstandings, especially with loved ones who may feel hurt or distant, this study found. Some people with HD tried to hide difficult feelings at work, or around new friends, but this often made them to more frustrated at home. Many people with HD reported cancelling plans with friends and spending more time alone, to protect themselves and others. 

Soothing the Struggle

During the interviews, participants shared different methods they used to keep irritability from boiling over. Taking breaks, distracting themselves, or leaning on supportive friends and professionals were common strategies. Alternatively, some suggested that medication and small lifestyle changes, like avoiding stressors, helped them to stay calm and protect their peace.

A Call for Compassion

Studies like this document in the scientific literature what many people from HD families already know. This type of research is critical so that patient organisations, clinicians and other HD stakeholders can lobby for resources, support services, and treatment strategies that address the full spectrum of HD symptoms. HD is more than just movement issues; it involves emotional, cognitive, and behavioral challenges that can deeply affect quality of life. Recognizing and validating these symptoms through research ensures they are not overlooked in care plans or drug development efforts.

If you are living with HD or caring for someone who is, know that you are not alone. Irritability is not a sign of weakness or bad character. It’s a real and painful part of HD. With understanding, patience, and the right strategies, it can be managed.

Just like you would make room for someone using a wheelchair, or help someone with a broken leg, people with HD need compassion for the challenges you can’t always see, like irritability. Let’s look past the surface and recognise the storm for what it is. Irritability is a symptom, not the whole person.

TL;DR

• Irritability in HD is more than just a bad mood; it’s a real, distressing symptom that can feel like a sudden storm, affecting both the person with HD and those around them.
• A UK-based study led by Dr. Sarah Gunn explored how people with HD experience and manage irritability. Through interviews, researchers identified key triggers (like stress, fatigue, or chaotic environments), emotional and social challenges, and coping strategies (like taking breaks or seeking support).
• The study emphasizes that irritability is a valid, often invisible part of HD that deserves understanding, compassion, and targeted support in care and treatment.

Learn more

Original research article, “‘It’s more than just irritability’: perspectives and experiences of irritability among people affected by Huntington’s disease” (open access).

Welcome to Brainland, the bustling, 24/7 theme park in your head. There’s Memory Maze, Logic Log Flume, and of course, the Emotion Coaster, where your brain races through tracks themed around happiness, sadness, and anger. However, for some people who carry the gene for Huntington’s disease (HD), some of these rides start acting up long before the big attractions like Movement Mountain or Memory Maze show signs of trouble. Strangely, even when everything else seems fine, the Emotion Coaster may begin to stall, especially on the tracks of anger, sadness, and fear. 

This isn’t a theme park glitch; it’s real science, explored by Dr. Shahin Nasr and his team at Massachusetts General Hospital and Harvard Medical School. 

When the Emotion Coaster Goes Off Track

Most of us don’t think twice about reading someone’s face. A smile reflects happiness. A furrowed brow? Maybe anger. It’s automatic, like muscle memory because our brains are wired to pick up on emotional signals. But what happens when the brain can’t understand how others feel?

Reading facial expressions is crucial because it helps us navigate daily social life. It helps us understand how others feel, respond with empathy, and avoid misunderstandings. Whether it’s noticing a friend’s sadness behind a smile, picking up on a colleague’s confusion in a meeting, or sensing when to give someone space, facial cues guide how we connect, communicate, and make social decisions every single day. 

When this ability fades, as it can in HD, relationships can suffer. This is not because people with HD don’t care, but because they may struggle to understand emotional expressions in other people, a skill which many take for granted.

Buckle Up for the Emotion Tracks

Dr. Shahin Nasr and his team ran an important study on emotion recognition in people with pre-manifest HD. ‘Pre-manifest’ HD refers to people who have tested positive for the gene that causes HD (CAG number is 40 or more), but they do not yet display any recognisable symptoms (equivalent to HD Integrated Staging System (HD-ISS) Stage 0). 21 people with pre-manifest HD and 16 people who did not have HD were recruited in this study. 

Each participant was shown 70 different photographs of people. These photographs displayed different facial expressions reflecting different emotions, including happiness, anger, sadness, fear, surprise, disgust, and ‘neutral’ faces. Participants were provided with name cards for each emotion. The task was simple, choose one name card that best identifies the emotion being shown in the photograph.

Interestingly, the participants with pre-manifest HD were less accurate at recognising anger, sadness, and fear from facial expressions displayed in the photographs compared to the control group. These early struggles to recognise particular emotions from other people’s faces suggests that even during the pre-manifest stage of HD, the inner ability to read negative emotions (anger, sadness, and fear) may already be decreasing.

Under the Hood of the Emotion Coaster

To dig deeper, a smaller sub-group of participants had their brains scanned using functional MRI (fMRI) while they looked at images of faces showing different emotions (neutral, happy, and angry). 

Think of the brain scans like a theme park map that shows which rides are busiest. But instead of tracking people, it shows which parts of the brain are most active, based on where the blood flows. Dr. Nasr and his team wanted to see what was happening in the brain while participants looked at faces reflecting different emotions. Specifically, how busy the Emotion Coaster was in people with pre-manifest HD (meaning the brain area that helps us understand facial expressions in other people).

When this ability fades, as it can in HD, relationships can suffer. This is not because people with HD don’t care, but because they may struggle to understand emotional expressions in other people, a skill which many take for granted.

Interestingly, there is one particular region of the brain that helps us to understand what someone is feeling or thinking by looking at their face, body, or tone of voice. However, in people with pre-manifest HD, this area of the brain showed reduced activity. This wasn’t just about having difficulty seeing faces clearly or understanding faces in general. It was more like the Emotion Coaster wasn’t working properly, making it harder for people with pre-manifest HD to understand emotions through facial expressions in other people.

Fixing the Emotion Coaster Before It Breaks Down

These findings are important because they show that small changes in the brain can happen early, even before movement symptoms begin. This can affect how people with pre-manifest HD understand emotions through facial expressions in other people.

However, this isn’t just something interesting to note. If people with pre-manifest HD have trouble reading emotions, it can cause big problems in relationships, work, and mental health. They might not react the right way in tense situations, which can lead to fights, feeling alone, or more stress.

This research gives us hope. By spotting early warning signs, such as changes in brain activity, we may be able to better support emotional and cognitive health, in people with pre-manifest HD before other symptoms appear.

End of the Ride

HD doesn’t just appear overnight, it starts gradually, with little changes that may be easy to miss. This study highlights that one of the earlier signs of HD might be in how we understand facial expressions in other people. For example, spotting a frown, a worried look, or a hint of anger.

Dr. Nasr and his team confirmed that a specific part of the brain which is important in understanding emotions by looking at facial expressions, was not as active in people with pre-manifest HD. This is equivalent to spotting a problem on the Emotion Coaster, before it fully breaks down.

It is important to remember that this study is more than just science, it’s a step towards changing lives for people with HD. By spotting very early brain changes linked to emotion recognition, this is not just identifying a faulty ride, it is uncovering a potential early warning sign of HD. The earlier we see the signs, the sooner we can act. It also provides us with a chance to support the daily interactions for people with pre-manifest HD, protect their relationships and even their quality of life.

TL;DR

• Emotion Coaster Starts to Wobble Early – Even before classic HD symptoms like movement or memory issues appear, people with the gene may struggle to recognise facial expressions showing anger, sadness, or fear.
• Study in Pre-manifest HD Shows Reduced Accuracy – In a study by Dr. Shahin Nasr, participants with pre-manifest HD were less accurate at identifying negative emotions in photos of facial expressions, suggesting early emotional processing changes.
• Brain Scans Reveal Decreased Activity – fMRI scans showed that brain regions involved in recognising others’ emotions are less active in people with pre-manifest HD, even though they had no visible symptoms.
• Real-Life Impact – This difficulty reading emotions can affect relationships, cause misunderstandings, and increase stress, not due to a lack of empathy, but reduced ability to interpret social cues.
• Early Detection = Early Support – Spotting these changes could help clinicians intervene earlier to support emotional wellbeing, social functioning, and quality of life before HD symptoms fully develop.
• Hope on the Horizon – This research adds to our understanding of HD progression and offers a potential early marker for future interventions targeting emotional and cognitive health.

Learn More

Original research article, “Are you angry? Neural basis of impaired facial expression recognition in pre-manifest Huntington’s” (open access)

Huntington’s disease (HD) is caused by a single genetic mutation, yet people with HD can experience vastly different symptoms – from movement issues to emotional struggles. A new study dives into brain connectivity to explore why that might be. Using MRI scans and thinking, movement, and behavioral tests, researchers identified two major clinical patterns and linked them to unique brain wiring signatures. These findings could pave the way toward more personalized HD care, tailored to an individual’s specific brain changes.

Same Cause, Different Routes

HD is caused by a CAG repeat expansion in the HTT gene. You’d think that if the genetic cause is the same, the disease might take the same route for everyone. But that’s not the case. People with HD show an incredible variety of symptoms: one person might struggle with balance and coordination, while another has emotional outbursts or depression, and a third faces memory challenges.

This diversity makes HD tough to understand, like trying to fix a car when each driver reports completely different problems – brakes in one, engine in another, electronics in a third. So scientists have been asking: What’s behind all this variability? A new study takes a close look at how the brain’s wiring – its functional connectivity – might hold the key.

We can think of the brain as a city with neighborhoods (regions) connected by roads (neural pathways). When traffic patterns change – some roads get blocked while others reroute – it can lead to very different commutes. That’s essentially what this study explored: how traffic in the brain changes in people with HD, and how those changes might explain the diverse ways the disease shows up.

Navigating the Symptom Patterns

To tackle this question, researchers collected lots of data from people with the HD gene. Participants underwent detailed testing: motor assessments (like walking and balance), cognitive evaluations (such as word fluency and mental flexibility), and behavioral surveys capturing mood, anxiety, and motivation. On top of all that, they had brain scans – specifically, structural and functional MRIs.

But how do you make sense of that mountain of data? Enter principal component analysis, or PCA – a mathematical tool that looks for themes in complex information. Imagine PCA as Marie Kondo for data: it finds the few key patterns that “spark the most variation” and tidies up the rest.

The analysis divided the data to uncover two distinct clinical profiles. The first, called the motor-cognitive profile, combined movement problems with thinking difficulties. The second, the behavioral profile, was characterized by mood and motivation issues – things like apathy, depression, and anxiety. It’s as if HD can take two different routes, depending on which brain circuits are most affected.

The analysis divided the data to uncover two distinct clinical profiles. The first, called the motor-cognitive profile, combined movement problems with thinking difficulties. The second, the behavioral profile, was characterized by mood and motivation issues.

Mapping the Motor-Cognitive Profile

Let’s zoom in on the motor-cognitive group. The brain scans revealed a fascinating, and somewhat unexpected, pattern. Certain connections were weaker, particularly between deep brain areas like the dorsal striatum (which includes the caudate and putamen that are brain region that experience the most cell loss in HD) and key regions of the cortex involved in planning and decision-making, like the dorsolateral prefrontal cortex.

This makes sense: if the brain’s “motor headquarters” can’t coordinate with the “executive offices” that plan and control actions, both movement and thinking take a hit. But here’s the twist – some connections in this group were stronger. Specifically, the ventral striatum (especially the nucleus accumbens, a region linked to motivation and reward) showed increased communication with those same executive areas.

Why would some brain pathways go quiet while others get louder? Researchers believe this may be a kind of internal rescue mission. As the usual routes break down, the brain may reroute traffic through better-preserved detours. This could be the brain’s way of compensating – trying to preserve function by strengthening alternate circuits. It’s like a city opening up a side street when the main highway is under construction.

Even more compelling: people with smaller volumes in the caudate and putamen (signs of more degeneration) showed greater activity in these compensating circuits. It’s as if the more damage there was, the harder the surviving systems worked to adapt.

Mapping the Behavioral Profile

The story was different for the second group – the behavioral profile. These individuals didn’t show the mix of weak and strong connections seen in the motor-cognitive group. Instead, they had a more uniform decrease in connectivity, especially between the putamen and parts of the limbic system – a network crucial for emotion, memory, and social behavior.

Key disrupted connections included links to the perihippocampal gyrus, involved in contextual memory, and the orbitofrontal cortex, a region critical for emotional regulation and reading social cues. This aligns perfectly with the types of symptoms seen in this group: trouble with mood, motivation, and behavior.

It’s as if the communication lines between emotional centers and control centers are down, making it harder for these individuals to manage emotions, maintain motivation, or engage socially. These patterns mirror findings in depression and apathy even outside of HD, strengthening the idea that the behavioral symptoms in HD may stem from similar wiring problems.

By pinpointing where the network disruptions occur, this study adds weight to the idea that HD isn’t just a disease of isolated brain regions – it’s a disease of circuits.

Two people with similar gene mutations might have different symptoms because their brains are rewiring differently. And that opens the door to more personalized care.

Toward Personalized Care

So what does all this mean for people living with HD? It shows us that how the brain is affected – not just how much – matters. Two people with similar gene mutations might have different symptoms because their brains are rewiring differently. And that opens the door to more personalized care.

Imagine using brain scans as a sort of GPS, mapping out which circuits are faltering or compensating. If a patient’s scans show signs of a motor-cognitive profile, care teams might focus on cognitive rehabilitation or motor training. If a behavioral profile is more prominent, mood-focused interventions could take center stage.

This approach could also reshape clinical trials. Rather than testing a drug across all people with HD, or even people with HD at a certain stage, researchers could see if it helps a specific subgroup – say, those with emotional disruptions or those relying heavily on compensatory circuits. That would make it easier to tell if a treatment is really working where it’s needed most.

Of course, there are caveats. The MRI scans in this study weren’t ultra-high-resolution, and the analysis didn’t include people without the HD gene. Plus, the researchers acknowledge that there may be more than two profiles out there; future studies with larger and more diverse samples might uncover even more patterns.

Still, the message is clear: HD is not a one-size-fits-all disease, and treating it shouldn’t be one-size-fits-all either. By listening closely to the brain’s wiring, scientists are getting closer to crafting treatment plans as unique as the people they’re meant to help.

TL;DR Summary

• Same gene, different symptoms – HD stems from one mutation but leads to varied issues like movement, mood, or memory problems.

• Two main profiles found – Researchers identified motor-cognitive and behavioral symptom patterns.

• Motor-cognitive profile – Weakened links between movement and thinking areas, with some stronger “compensating” circuits.

• Behavioral profile – Reduced connectivity in emotion and motivation networks, especially involving the limbic system.

• Brain wiring shapes symptoms – Differences in circuit disruption may explain why HD looks different in each person.

• Toward personalized care – Findings support tailored treatments and trials based on brain connectivity patterns.

Learn More

Original research article, “Striato-cortical connectivity patterns predict clinical profiles in Huntington’s disease”. (open access)

We received an update on June 2, 2025 from uniQure about their recent discussions with the US Food and Drug Administration (FDA) regarding the development of AMT-130 – a treatment they’re testing for Huntington’s disease (HD). uniQure announced that they remain aligned with the FDA and have received guidance on next steps, including plans for manufacturing, statistics, and a comparison control group. So what exactly did we learn from this recent update? Let’s get into it!

Laying the Tracks

We’ve been following the development of uniQure’s HTT-lowering drug, AMT-130, from its genesis in the lab, to animal studies just a few years ago, and now through to their ongoing Phase 1/2 clinical trials. 

We’ve also written about it quite a bit. To save our regular readers from repetition, we’ll link any HDBuzz newbies to previous news we’ve covered:

• Promising results from animal models, covered in April, 2021
• The announcement of the clinical trial, covered in July, 2021
• 12 month safety data, covered in July, 2022
• A short pause in the trial, covered in August, 2022
• The trial resumes, covered in November, 2022
• A promising safety profile, covered in December, 2023
• AMT-130’s potential to slow clinical progression, covered in July, 2024
• Previous alignment with the FDA, covered in December, 2024

FDA Meetings

On June 2nd, CEO Matt Kapusta shared that uniQure had two milestone meetings, called Type B meetings, with the FDA. These are formal meetings between the regulatory agency and drug companies to discuss development and advancement of a medicine. Any face-to-face meeting with the FDA is a good opportunity for a drug company, because it helps to ensure that they’re moving in a direction that the agency supports. 

During these meetings, uniQure aligned with the FDA on a plan for submitting a Biologics Licensing Application (BLA). This is the formal application a drug company puts together to request permission to market a biologic, a type of therapy derived from living organisms, such as proteins, cells, or genetic material. Unlike traditional small-molecule drugs, biologics are typically larger and more complex, and include treatments like antibodies and gene therapies. UniQure’s Chief Medical Officer, Dr. Walid Abi-Saab, shared that the FDA agreed that data collected from their ongoing trials could be used to support a future BLA. In other words, uniQure may not need to run another trial before seeking accelerated approval for AMT-130. 

The most exciting part? UniQure shared that their hope is to submit this BLA for accelerated approval during the first quarter of next year – as soon as the end of March, 2026. BUT, that doesn’t mean this is a done deal. The upcoming data, anticipated by the end of September 2025, still has to show that the drug is having the expected effect, or else the FDA won’t approve the BLA. 

For serious conditions like HD, which carry a tremendous burden with limited treatment options, regulators may greenlight earlier access to drugs that show potential.

Approval Paths

As HD drugs advance through the clinic, we’ve been hearing hopeful news about “accelerated approval.” But what exactly does that mean, and how does it differ from traditional approval?

First, the easy one – traditional approval. This means that a drug has undergone rigorous testing, is safe and well tolerated, and has improved symptoms or delayed worsening of a disease. Drugs that gain traditional approval have lots and lots of data to support their use, and they are very unlikely to be pulled from the market.  

With accelerated approval, a regulatory agency allows a drug to be made available to the general population while the drug is still being tested. There isn’t enough data yet to conclusively say that it’s having the desired effect. However, for serious conditions like HD, which carry a tremendous burden with limited treatment options, regulators may greenlight earlier access to drugs that show potential. 

The idea is that the accelerated approval path could help people in need access drugs sooner, in hopes that they will work. But drugs made available through accelerated approval could still be pulled from the market if additional testing suggests that they don’t actually benefit patients. While no one wants to take drugs that don’t work, many people accept the risk of taking drugs that might work, which is why uniQure is exploring the accelerated approval path. 

Regulatory milestones

To make sure that uniQure and the FDA are aligned on steps towards a speedy (possible) approval of AMT-130, they talked about a few key points: 

Manufacturing

Thinking ahead, the FDA wants to know if uniQure is capable of manufacturing  AMT-130 at scale for people with HD, should the data continue to look good. UniQure shared that the FDA agrees they have the manufacturing capabilities and has endorsed their production platform. This streamlines uniQure’s path toward commercial readiness and  a future BLA submission. 

Statistical Plan

During the meeting, the FDA and uniQure also talked about the type of data they want to see and how those data should be analyzed. Previously, the FDA agreed that positive data for the composite Unified Huntington’s Disease Rating Scale, or cUHDRS, could be used to decide whether the drug is working. They also discussed a  biomarker called NfL, which increases as HD progresses. They agreed that if AMT-130 reduces NfL, it could be  taken as a positive sign that the treatment is working.

This is exciting news because it sets the bar for what needs to be achieved to have the FDA approve a drug for HD. UniQure’s announcement confirmed that the FDA is still in support of using these metrics to help decide whether an HD drug is working. 

They’ve also agreed on exactly what types of statistical tests should be used to analyze the data. It’s great to know that the FDA and uniQure are aligned on the nitty gritty, but we’ll spare you those mathematical details here! 

Control Group 

Another important point the agency agreed on was which dataset uniQure should use to compare their trial results. While the first cohort included people who received a mock surgery, the ongoing AMT-130 studies don’t include a traditional placebo group of participants who did not receive the drug. Since this a long-term study being conducted over several years, it was instead designed to compare people receiving AMT-130 to participants in a natural history study. 

In a natural history study, no drug is given. It’s designed to follow people who have the gene for HD and to observe them as they naturally live and age to see how HD progresses. These types of studies give us a wealth of information.

UniQure were previously comparing AMT-130 recipients to those who participated in the TRACK-HD, TrackOn-HD, and PREDICT-HD studies. They’re now also incorporating data from Enroll-HD – the world’s largest observational study for HD families. The Enroll-HD dataset will be used for the primary analysis, and data from TRACK-HD and TrackOn-HD will be used for additional analysis. 

This change was made because the Enroll-HD dataset is significantly larger, at almost 33,000 people enrolled (wow!), many of whom fall into the inclusion criteria for the AMT-130 trials. This gives a better comparator when looking at the data and increases the chances of seeing a stronger effect of AMT-130.

They’ll be presenting this data publicly during the third quarter, so we should have a much better idea of AMT-130’s 3-year effectiveness by the end of September, 2025. After that, they plan to meet again with the FDA in the fourth quarter of 2025, to prepare for submitting their BLA in the first quarter of 2026.

The Track Ahead

On the June 2nd investor call, uniQure gave us a date for when they would stop collecting data to send over to the FDA – June 30. That cutoff will include data from people who were given AMT-130 3 years ago, so it’s quite a long-term follow up. 

They also shared that they’ll be presenting this data publicly during the third quarter, so we should have a much better idea of AMT-130’s 3-year effectiveness by the end of September, 2025. After that, they plan to meet again with the FDA in the fourth quarter of 2025, to prepare for submitting their BLA in the first quarter of 2026. At that meeting, uniQure will request that the FDA make their review a priority.

To continue laying the track to reach an HD gene therapy, the data must continue to be positive. In the meantime, we’ll be standing on the platform, waiting to let you know when the whistle blows!

TL;DR Summary

• A recent meeting between the FDA and uniQure continues to show alignment
• cUHDRS and NfL lowering continue to be agreed upon metrics for success
• UniQure got a green light from the FDA on their manufacturing platform
• Statistical plans for data analysis were discussed and agreed upon
• Enroll-HD will be used as the natural history control group for primary analysis
• Next data update – Q3 2025, which will include 3 year follow up data
• Q4 2025 meeting with the FDA to discuss accelerated approval
• Plan to submit Biologics Licensing Application for accelerated approval in Q1 2026

Learn More

UniQure’s June 2, 2025 webcast.

UniQure’s June 2, 2025 press release.

We’re pleased to announce the 2025 HDBuzz Prize! This year, the HDBuzz Prize is brought to you by the Hereditary Disease Foundation (HDF), who are sponsoring this year’s competition. Training the next generation of passionate Huntington’s disease (HD) researchers is directly in line with the HDF’s mission. The HDF understands the importance of having future key opinion leaders that can communicate their work to the global HD community in easy-to-understand language. They aim to ensure that we continue to have an abundant pipeline of talented researchers focused on HD – something we can all agree is critical for advancing HD research!

What Is The HDBuzz Prize?

The HDBuzz prize is an opportunity for early-career HD researchers to get involved in communicating HD science to the global community, see their work published and shared through our online channels, and win US $200 – woohoo for you! 

The prize was designed to diversify the voices that bring you content on HDBuzz. Not every researcher interprets data the same, so having multiple viewpoints represented here helps ensure that our readers are getting HD news content that spans the varied perspectives of the HD field.

Calling All HD Scientists

We’re looking for young scientists with a gift for communicating research news clearly and imaginatively.

The HDBuzz prize is open to anyone with an active involvement in any aspect of Huntington’s disease research. We’re particularly keen on the fresh take that PhD students and postdocs provide and are actively seeking researchers with clinical experience. So if that’s you, hit us up!

Full articles will include a piece of around 1,000 words, suitable for publication by HDBuzz, about a recent HD research paper that we haven’t covered yet.

In addition to the article, full article submissions will contain:

• a catchy title
• a front-page summary of no more than 100 words
• a social media post of no more than 250 characters, including spaces
• two in-text image suggestions and a cover photo suggestion
• at least one reference to a peer-reviewed article forming the subject of the piece

The HDBuzz prize is an opportunity for early-career HD researchers to get involved in communicating HD science to the global community, see their work published and shared through our online channels, and win US $200 – woohoo for you!

How To Apply

Entrants should submit a short pitch, of ~200 words detailing what they wanted to write about and why they thought it was important for HD families to know about, all in HDBuzz-style language. We will review all of the article pitches to screen ideas and writing styles before inviting full articles to be written. Our goal is to save everyone time! We want HD researchers to be able to spend as much time working on their science.

The closing date for entries for the article pitches is Tuesday 1st July at 5pm Eastern Standard Time.

All entries must be written in English. We regret that entries in other languages will not be considered.

Prizes!

The pitch winners will be announced throughout the Fall (or autumn, if you’re feeling fancy – or just British to be honest) of 2025.

Winning articles will be published on HDBuzz, translated into various languages, and syndicated to dozens of HD community sites worldwide.

Winners will also receive US $200!

Public engagement is important for every scientist’s training, so apart from the cash, this would enhance the CV of any eager young HD researcher.

Tips For Successful Writing

1. Take a look at several HDBuzz articles before you start, to get a good feel for our tone and style.
2. If in doubt, simplify.
3. Assume that your reader is interested in HD and willing to learn, but has no formal scientific training.
4. Explain anything beyond high-school science, in bite-sized steps. Then go back and explain the high-school science.
5. Apply metaphor, analogy, humor, and silliness generously.
6. Remember to explore the limitations of what you’re writing about and try to explain what needs to happen next for it to bring HD treatments closer.
7. Avoid pictures that contain text for full article submissions, as this causes translation problems.
8. Avoid studies we’ve already covered, publications with which you’re affiliated, and any pre-print articles.

The Fine Print

The editors’ decision is final and correspondence will not be entered into. By submitting an entry, you are agreeing to allow editing of your article for style and content, and its publication via HDBuzz.net and release under the Creative Commons Attribution-ShareAlike 3.0 Unported License including translation and unlimited syndication. Winners agree to be named at HDBuzz.net. All entries will be scrutinized for plagiarism and disqualified if it is found. 

Public engagement is important for every scientist’s training, so apart from the cash, this would enhance the CV of any eager young HD researcher.

Help Us Spread The Word!

Whether you’re a Principal Investigator (that’s ‘head honcho’ in laboratory-speak) or an HD family member, chances are you know a young scientist with a neat turn of phrase and a gift for communication.

If so, please put them in touch with us or send them to this article, so they can think about entering the prize.

So, fellow science nerds – get in touch and get cracking!