Credits: Canva
Huntington disease (HD) has finally found its first successful treatment. Biotech company uniQure has announced positive top-line results from its Phase I/II trial of AMT-130.
It is a one-time gene therapy for HD. According to the update, the therapy significantly slowed symptom progression and met its primary endpoint, making it the first drug ever to alter the course of HD in a human clinical trial.
Topline data refers to an early summary of key results from a study. In this case, the findings suggest that AMT-130 may meaningfully delay disease decline across movement, thinking, and daily functioning.
Huntington’s disease is a rare, inherited neurodegenerative disorder caused by a faulty copy of the huntingtin gene. This gene contains an abnormal expansion of the DNA sequence CAG, which leads to production of a toxic form of the huntingtin protein. Over time, this protein damages brain cells, causing movement problems, cognitive decline, and loss of independence.
Currently, there are no approved treatments that can slow or stop the progression of HD—existing drugs only manage symptoms. This makes the results from AMT-130 especially significant.
Unlike pills or injections that require repeated dosing, AMT-130 is a one-time gene therapy delivered directly into the brain through surgery.
The therapy uses a harmless virus (AAV5) as a delivery vehicle. This “Trojan Horse” carries genetic instructions that bind to the messages cells normally use to make the huntingtin protein. By marking these instructions for destruction, AMT-130 reduces the overall levels of huntingtin protein—including the harmful version linked to HD.
uniQure believes this could provide a long-lasting or potentially lifelong effect, distinguishing it from other huntingtin-lowering drugs in development.
Gene therapies carry risks, especially when delivered via brain surgery. Initial stages of the trial were cautious, with small participant groups and strict safety monitoring. Some patients experienced serious side effects early on, prompting temporary pauses and safety reviews.
Despite these hurdles, trial participants, the first humans ever to receive AMT-130—took a historic step forward. Their involvement paved the way for further testing, and by mid-2024, interim updates began showing encouraging trends. Biomarkers of brain health improved, disease progression appeared slower, and no major new safety concerns were reported.
The topline results released this week provide the clearest picture yet of AMT-130’s potential. Researchers compared participants’ outcomes to data from Enroll-HD, a large database tracking the natural progression of the disease. The findings suggest substantial slowing of decline in patients receiving the high dose of AMT-130.
This combined score tracks movement, thinking, independence, and daily function. Patients on high-dose AMT-130 showed a 75% slowing of decline compared to controls. In practical terms, a decline expected in one year could now take four years.
TFC assesses real-world abilities such as managing finances, working, and living independently. AMT-130 slowed decline in TFC by about 60%, reinforcing the cUHDRS findings.
In the Symbol Digit Modality Test (SDMT), which measures mental processing speed, AMT-130 slowed decline by 88%, though the result narrowly missed statistical significance. Another test, the Stroop Word Reading Test (SWRT), showed an impressive 113% slowing, suggesting potential benefits for attention and language.
Using the Total Motor Score (TMS), which evaluates involuntary movements and coordination, AMT-130 showed a 59% slowing in symptom worsening. While this result was not statistically significant, it suggests possible benefit across motor symptoms.
Neurofilament light (NfL), a protein released when brain cells are damaged, typically rises in HD patients. At 36 months post-treatment, high-dose AMT-130 patients had an 8% drop in NfL levels—suggesting reduced ongoing brain cell injury.
Overall, AMT-130 was well tolerated. No new serious drug-related side effects have been reported since late 2022. Most issues were linked to the surgical procedure itself and have since resolved.
The difference in outcomes between high-dose and low-dose groups underscores the importance of dosing strength, with the high-dose cohort showing the most consistent benefit.
Following these results, uniQure plans to meet with the U.S. Food and Drug Administration (FDA) later this year. The company aims to file for a Biologics License Application (BLA) in early 2026. If successful, AMT-130 could be launched in the U.S. later that year.
Discussions are also planned with European regulators, including the European Medicines Agency (EMA). Meanwhile, ongoing studies will continue to treat more participants, including those at different stages of HD who were previously ineligible.
Cancer deaths in the United Kingdom have dropped to their lowest recorded levels, according to new data from the charity Cancer Research UK. The figures show that cancer death rates have fallen by 11 per cent in the past decade, reflecting progress in early detection, screening, treatment and prevention.
Researchers estimate that around 247 people in every 100,000 in the UK now die from cancer each year. This is a significant decline from the peak recorded in 1989, when about 355 people per 100,000 died annually from the disease. Overall, that represents a 29 per cent reduction over the past few decades.
Experts say this steady improvement is the result of sustained scientific progress, improved healthcare systems and public health measures that target risk factors such as smoking.
The new data highlights falling death rates across several major cancers. Ovarian cancer deaths have dropped by 19 per cent over the past ten years, up to 2024. Lung cancer deaths have fallen by 22 per cent during the same period, reflecting the long-term impact of reduced smoking rates and better treatment options.
Deaths from stomach cancer have seen one of the most dramatic improvements, dropping by 34 per cent in the past decade. Bowel cancer deaths have decreased by six per cent, while breast cancer deaths have fallen by 14 per cent.
Other cancers have also seen notable declines. Cervical and prostate cancer deaths have both dropped by 11 per cent. Deaths from leukemia are down by nine per cent, while esophageal cancer deaths have fallen by 12 per cent.
Experts say improved diagnosis, new therapies and better awareness are playing a key role in these trends.
One of the biggest public health successes has been the decline in cervical cancer deaths. Since the 1970s, deaths from cervical cancer have fallen by around 75 per cent in the UK. Health experts attribute much of this progress to the national cervical screening programme run by the NHS.
Screening helps detect abnormal cells early, allowing treatment before cancer develops or spreads.
Another major contributor is the human papillomavirus vaccine, commonly known as the HPV vaccine. The vaccine protects against the virus responsible for most cervical cancer cases. It is routinely offered to schoolchildren in the UK, and since its introduction in 2008, at least 6.5 million young people have received it.
Public health experts believe the vaccine will continue to reduce cervical cancer rates in the coming decades.
Despite the overall progress, the data also shows worrying increases in deaths from certain cancers. Gallbladder cancer deaths have risen by 29 per cent, while deaths from eye cancer have increased by 26 per cent.
Liver cancer deaths are up by 14 per cent, and kidney cancer deaths have risen by five per cent. Meanwhile, death rates for thyroid cancer, pancreatic cancer and melanoma have remained largely unchanged.
Another important trend is that the total number of people dying from cancer continues to rise. This is largely due to population growth and an ageing population, as cancer risk increases with age.
Experts say the long-term decline in cancer deaths reflects decades of medical research and innovation. However, they stress that continued investment is necessary to sustain progress.
Cancer Research UK researcher Dr Sam Godfrey said the figures show the impact of scientific breakthroughs over many years. He has called on the government to support more clinical trials and ensure that NHS staff have enough time and resources to take part in life saving research.
Public health policies such as smoking bans, along with screening programmes and vaccines, are also credited with helping drive down cancer deaths across the country.
Credit: Canva
Are you in the habit of catching up on social media or news updates while sitting on the pot? A new study shows you may be "unintentionally" spending extra time and increasing your risk of developing painful hemorrhoids by 46 percent.
The study, published in the open-access journal PLOS One, explained that getting distracted by news or social media can increase pressure on sensitive anal tissues, which leads to hemorrhoids.
Hemorrhoids, also called piles, are swollen and inflamed veins around your anus or in your lower rectum.
"Using a smartphone while on the toilet was linked to a 46 percent increased chance of having hemorrhoids. We're still uncovering the many ways smartphones and our modern way of life impact our health,” Trisha Pasricha, from the Beth Israel Deaconess Medical Center in the US.
“It's possible that how and where we use them -- such as while in the bathroom -- can have unintended consequences," she added.
For the study, the researchers examined data from colonoscopies of 125 adults in America, and conducted an online survey to understand their lifestyle habits and behavior while using the toilet.
Two-thirds of the participants reported using their smartphones while on
the toilet. Compared with those who did not, endoscopists found that they had a 46 percent higher risk of hemorrhoids.
More than a third of bathroom smartphone users reported spending more than five minutes there during a single visit -- reading news (54.3 percent), or browsing social media (44.4 percent).
In comparison, just 7.1 percent of non-users reported staying that long.
"Smartphone use may unintentionally extend the time people spend sitting on the toilet. Sitting for longer periods could increase pressure on tissues in the anal region, which may contribute to the development of hemorrhoids,” the researchers said.
Pasricha suggested individuals leave smartphones outside the bathroom to understand the actual time it takes for a bowel movement.
"If it's taking longer, ask yourself why. Was it because having a bowel movement was really so difficult, or was it because my focus was elsewhere?" she said, calling for more studies.
Studies estimate that hemorrhoidal disease affects 40% of people all over the world, and it is one of the most common diseases in the anorectal region.
The two types of hemorrhoids are:
Credits: X/Twitter
Iran's capital Tehran was engulfed in a black cloud of toxic smoke. This also unleashed a black rainfall on Sunday after overnight Israeli strikes on several fuel depots caused fires to burn for hours. Images have come from across the city of Tehran. These images show thick black smoke from the fires hanging over it. Residents have also reported difficulty breathing and oil-tainted rainfall staining everything around them.
As per a TIME report, Iran's Red Crescent Society warned the residents of Tehran and the surrounding region that the rainfall after the strikes could be "highly dangerous and acidic", and could cause "chemical burns of the skin and serious damage to the lungs".
Many have complained about breathing problems, along with headache, feeling dry and sore lips, and feeling like burn in the eyes and constant itch in the throat.
Iran's Red Crescent Society issued statements on Telegram that the rain could be contaminated with "toxic hydrocarbon compounds" as well as "sulfur and nitrogen oxides".
As per a report by The Conversation, people exposed to the black smoke in Iran could experience headaches or difficulty in breathing, especially if they have asthma or a lung disease.
People who are more prone to health issues are older people, young children, anyone with disabilities and pregnant women. This could also lead to lower birth weights.
Since the thick black cloud from all the burning could increase the PM2.5 or the ultrafine particles, known as particulate matter, it could also increase cancer risks, along with neurological conditions and cardiovascular conditions.

The toxic rain could further pollute the natural waterways and drinking water sources. A photo shared by Iran's Red Crescent shows a healthcare worker's uniform covered in black droplets from the rain.
The "rain drops" are tainted with oily residue and could lead to skin problems, and if inhaled, it could also lead to serious medical crisis, noted Jim NR Dale, a senior meteorologist at British Weather Services.
It may also carry carcinogenic polycyclic aromatic hydrocarbons (PAHs) along with heavy metals that are released when construction materials burn and then remain suspended in the air.
As acidity increases, natural water bodies such as rivers and lakes can become too hostile to support life. When the pH of water drops below 5, most fish cannot survive, and at pH 4, a lake is often described as a “dead water body” because almost no living organisms remain.
Acid rain also harms the soil. It reduces calcium levels, an important nutrient for plants, and makes it easier for toxic aluminium to leach into water sources, further threatening ecosystems.
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