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.
Credits: Canva
Norovirus, often called the “stomach flu” or “winter vomiting illness,” is currently circulating in California and across the United States. Health experts say proper cleaning and disinfecting can slow its spread. These precautions matter because norovirus is extremely contagious and there is no specific treatment for it, according to the Centers for Disease Control and Prevention.
Between August 1 and November 13, health officials recorded 153 norovirus outbreaks across 14 states. The CDC defines an outbreak as two or more similar illnesses linked to suspected or lab-confirmed norovirus cases. Although California is not among the states reporting outbreaks, CDC data shows a noticeable rise in positive norovirus tests throughout November across the western US.
Norovirus is described as a “highly contagious” virus that causes vomiting, diarrhea, and stomach cramps, according to the California Department of Public Health. It leads to acute gastroenteritis, which means inflammation of the stomach or intestines, and affects people of all ages, the CDC said.
Despite its nickname, norovirus has nothing to do with influenza. The flu is caused by the influenza virus, not norovirus, the agency clarified.
Norovirus spreads easily from person to person, especially while someone is actively sick, according to the California Department of Public Health. Symptoms usually last one to three days, but a person can still spread the virus for up to two days after they start feeling better.
The virus is present in vomit and stool and can spray into the air or settle on nearby surfaces. It can survive for weeks on uncleaned items such as countertops, phones, and doorknobs, the department said.
“You can get sick after touching or caring for someone who is sick, or after touching or eating something that is contaminated,” the department noted.
Norovirus spreads quickly in crowded or enclosed settings, including schools, child care facilities, hospitals, nursing homes, jails, and cruise ships.
According to state and federal health officials, the most common ways people catch norovirus include:
“If you or someone in your household has norovirus, it’s important to clean and disinfect areas where vomiting or diarrhea occurred,” the California Department of Public Health advised. Cleaning with soap and water removes germs, while disinfecting kills those that remain.
You can use commercial disinfectants, but it’s important to read the label and make sure norovirus is listed among the germs it targets. You can also check the Environmental Protection Agency’s registration number and confirm it appears on the EPA’s List G, which includes products proven effective against norovirus.
Another option is making a bleach solution at home. The US Department of Agriculture recommends mixing 5 to 25 tablespoons of household bleach with one gallon of water. This solution should be used only on hard or nonporous surfaces and applied with a spray bottle or disposable mop, sponge, or paper towel. Let it sit for about five minutes before wiping it away.
The USDA also advises paying close attention to frequently touched items such as doorknobs, handles, and remote controls. Many disinfectant wipes do not fully kill norovirus, the agency warned.
The CDC recommends wearing rubber or disposable gloves while cleaning. Use paper towels to wipe the affected area completely, then place the towels in a plastic trash bag. The California Department of Public Health also suggests wearing a face mask.
After disinfecting, clean the area again with soap and water, the CDC said. Keep in mind that vomit can aerosolize the virus, meaning surfaces several feet away may also be contaminated, according to the USDA.
Once you’re done, throw away the gloves and mask, then take out the trash, the California Department of Public Health advised.
Credits: Canva
Millions of people across the UK who take certain everyday medicines are being warned by the NHS to steer clear of a specific fruit. Health officials say combining this fruit or its juice with some medications can trigger serious reactions and unexpected side effects. The advice applies to patients prescribed some of the most commonly used drugs in the country, including certain statins, amlodipine, and medicines for anxiety. Statins, in particular, are widely prescribed to help reduce levels of “bad” LDL cholesterol in the blood.
Statins work by limiting how much cholesterol the liver makes. This helps slow down the build-up of fatty deposits inside the arteries, which in turn lowers the risk of heart attacks and strokes. Doctors prescribe statins both to manage high cholesterol and to prevent future heart-related problems. For most people, they are taken daily as tablets and are often part of long-term treatment.
Amlodipine, commonly sold under the brand name Istin, belongs to a group of medicines known as calcium channel blockers. It is mainly used to treat high blood pressure. For people with hypertension, amlodipine can reduce strain on the heart and lower the risk of serious complications such as heart disease, heart attacks, and strokes.
Patients taking certain forms of amlodipine, particularly calcium channel blockers, are advised not to consume grapefruit. The NHS website clearly states: “Do not take amlodipine with grapefruit juice.”
The reason lies in how grapefruit affects the body. It interferes with enzymes and transport proteins in the gut, such as CYP3A4 and P-gp, which normally help break down medicines and control how much enters the bloodstream. When these processes are blocked, higher-than-intended amounts of the drug can build up in the body, increasing the risk of side effects.
Not every medicine reacts the same way to grapefruit juice, which is why doctors advise checking before making changes. Always speak to your GP or pharmacist, read the information leaflet that comes with your medicine, and look out for any warnings related to grapefruit.
It is also important to understand that simply spacing the fruit and the medicine apart is not enough. In most cases, grapefruit should be avoided entirely for as long as you are on the medication, unless your doctor says otherwise. Patients are also encouraged to be cautious with other citrus fruits, such as Seville oranges, pomelos, and limes, as they can have similar effects on how medicines work.
If you have eaten grapefruit or had its juice while taking statins, amlodipine, or anti-anxiety medication, there is no need to panic. In many cases, a single exposure does not lead to serious harm, but it is important to stay alert. Pay attention to how your body feels over the next day or two, especially if you notice symptoms such as dizziness, muscle pain, unusual tiredness, nausea, flushing, or a sudden drop in blood pressure.
Do not stop taking your medication on your own. Skipping doses or stopping treatment abruptly can be risky, particularly for heart and blood pressure medicines. Instead, make a note of what you consumed and when, and contact your GP or pharmacist for advice. They can tell you whether any action is needed based on the specific drug, dose, and your overall health.
Credits: Canva
During the festive months, overindulgence in celebrations can trigger holiday heart syndrome (HHS), a potentially serious yet largely preventable set of heart-related symptoms that tend to rise during this time of year. Most people recover within a day, but HHS can occasionally lead to stroke, heart attack, or other serious complications, according to the Cleveland Clinic. As the holiday season approaches, let’s explore what holiday heart syndrome is, its symptoms, and how to prevent it.
First identified by a US physician in 1978, holiday heart syndrome describes irregular heartbeats that occur after binge drinking during the festive season. Research from the University of South Australia shows that alcohol consumption increases by 70 per cent in December compared to other months, while iSelect data predicts Australians may consume around 58 million standard drinks per day during the month. With numbers like these, some caution is warranted.
Atrial fibrillation (AFib) is the specific irregular heartbeat seen in HHS. It occurs when the heart’s upper chambers contract or quiver in a chaotic pattern, which can cause blood to pool in the atrium and form clots. If these clots enter the bloodstream, they can travel to the brain and trigger a stroke.
Symptoms can include fatigue, dizziness, shortness of breath, anxiety, weakness, confusion, sweating, faintness, unusual tiredness during exercise, rapid or irregular heartbeat, and sensations like fluttering, thumping, pressure, or chest pain.
Dr Mariann R. Piano, Nancy and Hilliard Travis Professor of Nursing at Vanderbilt University School of Nursing, warns, “The scary thing is that people may not notice any symptoms, which makes it a particularly dangerous arrhythmia.”
Researchers are still investigating exactly how alcohol triggers AFib and how much intake increases the risk. A 2020 study found that people who usually drank at least 10 alcoholic beverages a week lowered their risk of holiday heart syndrome by reducing alcohol consumption. Another study confirmed that eliminating alcohol significantly decreases the chances of developing AFib.
The Australian Alcohol Guidelines recommend that healthy adults limit alcohol to no more than 10 standard drinks per week to reduce risk.
Experts like Kistler suggest that people with existing heart arrhythmias either abstain or stick to “up to three standard drinks a week.” Drinking mindfully, reducing overall consumption, and staying well hydrated can also help protect your heart.
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