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In this phase 1 trial, treatment of patients with fibrolamellar hepatocellular carcinoma with a therapeutic peptide vaccine targeting the fusion kinase DNAJB1–PRKACA, which is the driver of the disease, together with nivolumab and ipilimumab, was safe and led to encouraging preliminary clinical responses, and translational analysis showed activation of immune responses.
A phase 1/2 and a phase 3 trial testing an AAV-FIX Padua gene therapy in patients with hemophilia B in China showed that the treatment was well tolerated and effective in reducing the annualized bleeding rate.
This study provides the first evidence in humans that tirzepatide modulates brain electrophysiology, with changes in low-frequency accumbens power linked to food preoccupation.
A combination of high-resolution spatial imaging, spatial proteomics and transcriptional data reveals sparse and heterogeneous bacterial signals in gliomas and brain metastases.
As presented at the American Heart Association Conference 2025, patients with chest pain and without obstructive coronary artery disease benefit from treatment informed by cardiovascular magnetic resonance.
As presented at AHA Scientific Sessions 2025, in a trial emulation analysis including benchmarking to data from randomized clinical trials, treatment with semaglutide and tirzepatide showed similar levels of benefit on cardiovascular outcomes in individuals at elevated cardiovascular risk with obesity and diabetes.
Elebsiran plus PEG-IFNα improved hepatitis B surface antigen (HBsAg) loss rates compared with PEG-IFNα alone in patients with chronic hepatitis B virus infection. Furthermore, prior response to the BRII-179 vaccine was associated with higher HBsAg clearance, suggesting its potential as a predictive tool for identifying patients more likely to benefit from therapies.
In a meta-analysis of 48 randomized trials of chronic kidney disease progression, reduction in the 6-month urinary albumin:creatinine ratio was associated with lower hazard ratios of established kidney disease endpoints, supporting the use of albuminuria change as a surrogate endpoint in clinical trials for chronic kidney disease.
Pretrained using 335,645 whole-slide images, a foundation model is developed to provide representations for slide- and patient-level tasks. It is capable of performing clinical tasks and generating reports even in data-scarce scenarios, such as rare cancer diagnosis and survival prediction, without requiring further fine-tuning.
In this follow-up study presenting results at 96 weeks, cabotegravir and rilpivirine long-acting therapy remained noninferior to oral therapy, had acceptable safety and tolerability profiles, and resulted in durable virologic suppression, further supporting its consideration for African treatment programs.
In cognitively normal older adults at risk for Alzheimer’s dementia, physical inactivity was associated with faster tau protein buildup and cognitive decline.
This study identifies four distinct polycystic ovary syndrome (PCOS) subtypes using unsupervised clustering analysis on data from 11,908 women and validated across five diverse cohorts. The subtypes show unique clinical features and suggest that subtype-specific management could enhance treatment precision for PCOS.
The biological clock model LifeClock predicts biological age across all life stages from routine clinical data, revealing distinct pediatric and adult disease risk patterns.
In a phase 1 dose-escalation study including 11 patients with heart failure who were followed for 12 months, delivery of a cardiotropic adeno-associated viral vector designed to deliver constitutively active protein phosphatase 1 inhibitor 1 to the heart was well tolerated and showed preliminary evidence of efficacy.
In a phase 1 trial, intramuscular injection of synthetic plasmid DNA encoding monoclonal antibodies against SARS-CoV-2 was safe and well tolerated and did not elicit antidrug antibodies.
Researchers analyzed 5,279 plasma proteins in 40 people undergoing feminizing gender-affirming hormone therapy over 6 months, revealing significant changes in 299 proteins. Such changes could have implications for reproductive capacity, immune regulation and long-term health.
Myelin damage in multiple sclerosis can be detected up to 7 years before symptoms, with early immune pathway activation and a 21-protein panel showing promise for presymptomatic diagnosis.
As presented at the ESMO Congress 2025: Results of the phase 2/3 AGITG DYNAMIC-III trial show that de-escalated chemotherapy based on ctDNA-negative status in patients with stage III colon cancer did not meet non-inferiority for 3-year recurrence-free survival when compared to standard of care, although it enables better informed treatment decisions.
