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A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.
FRIDAY, June 6, 2025 — A repurposed diabetes drug can be an effective tool against fatty liver disease, a new clinical trial has found. Dapagliflozin helped people reduce fat levels in their liver and avoid liver scarring that comes with the.
MONDAY, June 9, 2025 — Prompt treatment with a drug combo can effectively manage chronic kidney disease in people with type 2 diabetes, a new clinical trial shows. Patients prescribed the combination of finerenone and empagliflozin.
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FRIDAY, June 6, 2025 — A repurposed diabetes drug can be an effective tool against fatty liver disease, a new clinical trial has found.Dapagliflozin helped people reduce fat levels in their liver and avoid liver scarring that comes with the c.
The deal centers around a medicine that could “shift the treatment paradigm for cardiovascular disease,” Lilly said, but also follows a yearslong in decline in Verve’s share price amid skepticism over the need for such a therapy.
New hypothesis paper builds on a growing scientific consensus that Parkinson's disease route to the brain starts in either the nose or the gut and proposes that environmental toxicants are the likely source.
Related groups Xavier lab Over the past two decades, large genetic studies have linked tens of thousands of DNA variants to thousands of human traits and diseases. Yet, correcting the effects of those variants to treat disease has been hampered in part by the lack of precise molecular tools to do so.
A deal worth up to $712 million with the China-based biotech gives Cullinan T cell engagers aimed at BCMA and CD19, two targets of interest in inflammatory conditions.
A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design and test a new CRISPR medicine in just a few months.
Young to middle-aged women who reported drinking eight or more alcoholic beverages per week--more than one per day, on average--were significantly more likely to develop coronary heart disease compared with those who drank less, finds a study presented at the American College of Cardiology's Annual Scientific Session.
Higher exposure to these microplastics, which can be inadvertently consumed or inhaled, is associated with a heightened prevalence of chronic noncommunicable diseases, according to new research. Tiny fragments of plastic have become ubiquitous in our environment and our bodies.
THURSDAY, May 1, 2025 -- The cutting-edge weight-loss drug Ozempic/Wegovy can stop and even reverse fatty liver disease, a new clinical trial has found.Nearly twice as many people taking semaglutide stopped their fatty liver disease without further.
Interactions with friends and family may keep us healthy because they boost our immune system and reduce our risk of diseases such as heart disease, stroke and type 2 diabetes, new research suggests. Researchers drew this conclusion after studying proteins from blood samples taken from over 42,000 adults recruited to the UK Biobank.
Consuming moderate amounts of coffee and caffeine regularly may offer a protective effect against developing multiple cardiometabolic diseases, including type 2 diabetes, coronary heart disease and stroke, according to new research.
Research has revealed clear evidence that changes in the orchestration of brain oxygenation dynamics and neuronal function in Alzheimer's disease contribute to the neurodegeneration.
Unstable proteins are the main drivers of many different heritable diseases, according to a new study, including genetic disorders responsible for the formation of cataracts, and different types of rare neurological, developmental and muscle-wasting diseases.
Most treatments being pursued today to protect against Alzheimer's disease focus on amyloid plaques and tau tangles that accumulate in the brain, but new research points to a novel -- and noble -- approach: using Xenon gas. A phase 1 clinical trial of the treatment in healthy volunteers will begin in early 2025.
The findings indicate that concentrated physical activity patterns may be just as effective for disease prevention as patterns where exercise is spread out throughout the week.
A newly developed blood test for Alzheimer's disease not only aids in the diagnosis of the neurodegenerative condition but also indicates how far it has progressed, according to a new study.
An increase in physical activity between the ages of 45 and 65 could help prevent Alzheimer's disease, while inactivity may be detrimental to brain health.
16, 2024 -- Fatty liver disease linked to diabetes and obesity can easily progress to liver cirrhosis, but new research suggests that GLP-1 medicines like Ozempic can help stop that. MONDAY, Sept. In a new decades-long study, veterans with diabetes.
A medical breakthrough could result in the first treatment for rare but serious diseases in which genetic defects disrupt cellular energy production. Researchers have identified a molecule that helps more mitochondria function properly.
A bone marrow transplant process is safe and curative for adults with sickle cell disease, according to results of a trial completed at about 20 cancer centers.
Driven by an older, more diverse population, along with a significant increase in risk factors including high blood pressure and obesity, total costs related to cardiovascular disease (CVD) conditions are likely to triple by 2050, according to recent projections. At least 6 in 10 U.S. trillion price tag in direct and indirect costs.
Engineers have developed a pill that releases microscopic robots, or microrobots, into the colon to treat inflammatory bowel disease (IBD). The experimental treatment, given orally, has shown success in mice. It significantly reduced IBD symptoms and promoted the healing of damaged colon tissue without causing toxic side effects.
Over a ten-year period, biobank participants who met recommended levels of physical activity had a 23% lower risk of cardiovascular disease, and the protective effects were even more pronounced in individuals with depression.
Our organs age at different rates, and a blood test determining how much they've each aged could predict the risk of conditions like lung cancer and heart disease decades later, finds a new study.
Phase 3 results revealed Monday led shares to surge and should yield an approval application in IgA nephropathy, a competitive corner of drug research, later this year.
The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My
Boosting communication between the spinal nerves and the muscles using the spinal cord stimulation reverses spinal muscle atrophy (SMA) progression and could be applied to other motoneuron diseases, including ALS.
Low levels of traffic-related air pollution harms the liver and may raise the risk of metabolic-associated fatty liver disease, a new study in mice suggests.
Researchers have conducted one of the largest eye studies in the world to reveal new insights into retinal thickness, highlighting its potential in the early detection of diseases like type 2 diabetes, dementia and multiple sclerosis.
FRIDAY, April 4, 2025 -- An experimental blood test for Alzheimers disease not only can aid in its diagnosis but also indicate how far the brain condition has progressed, a new study says.The test tracks levels of a protein called MTBR-tau243, w.
THURSDAY, June 12, 2025 — Most folks tap away at their smartphone for fun, communication or information.But such tapping may also help them track the progress of a rare degenerative brain disease, researchers say.Five simple smartphone-measured t.
30, 2024 -- In the highest tally ever recorded for tuberculosis cases, the World Health Organization reported Tuesday that over 8 million people worldwide were diagnosed with the lung disease last year.Of WEDNESDAY, Oct. that number, 1.25
28, 2024 -- The weight-loss drug Ozempic can guard against kidney disease in obese people, a new study shows.Patients taking semaglutide -- the active agent in Ozempic and Wegovy -- had as much as a 52% reduction in kidney damage, as. MONDAY, Oct.
People who eat more ultra processed foods like cold breakfast cereal, cookies and hot dogs are more likely to have early signs of Parkinson's disease when compared to those who eat very few ultra processed foods, according to a new study.
THURSDAY, April 17, 2025 -- Common medications like aspirin, ibuprofen, statins and beta blockers appear to slow the progress of Parkinsons disease, a new study says.Parkinsons symptoms took nearly a decade longer to crop up in patients taking the.
A new study suggests that risk factors and biomarkers related to Alzheimer's disease are associated with cognition much earlier in life than previously recognized. The study highlights significant associations between cognition and Alzheimer's disease risk factors as young as ages 24 to 44 and underscores the importance of early prevention.
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