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Plozasiran reduced triglyceride levels by 80% and lowered the risk of pancreatitis in patients with persistent chylomicronemia, with or without a genetic diagnosis.

For individuals with pacemakers, a care pathway that includes echocardiographic screening to detect signs of heart failure did not improve cardiac outcomes, but patients flagged as having impaired heart function who were managed by a specialized heart failure clinic benefited, as compared to those managed by primary care physicians.

Studies in China show how large language models can improve primary healthcare systems, but equitably scaling this technology will require attention to rural, low-resource settings and the companion policies that support its implementation.

The ETER701 trial demonstrates that a four-drug regimen, involving the addition of anti-angiogenesis therapy to immuno-chemotherapy, improves survival outcomes for extensive-stage small-cell lung cancer — but is more indeed better when it comes to treating this intractable disease?

Genomic and epidemiologic analyses revealed that a novel reassortant viral lineage of the Oropouche virus, which has been circulating in the western Amazon region of Brazil for about a decade, is associated with the recent human outbreaks between 2022 and 2024.

As drastically rising global temperatures threaten the health and wellbeing of populations, solutions that drive policy action must be based on scientific evidence of which strategies work in different scenarios.

Extreme heat events are expected to become more frequent because of climate change. Our analysis of almost 140,000 births across 16 hospitals in four countries in sub-Saharan Africa indicates 34% higher odds of perinatal mortality (stillbirth or death up to 24 hours after birth) if extreme heat occurred in the week preceding childbirth.

Trained on a cohort of 45,064 cases and validated on data acquired from mobile computed tomography scanners deployed in rural China, a lung cancer screening deep learning model is shown to outperform existing lung cancer risk scores.

In a large cohort with a 23-month median follow-up of the CIRCULATE-Japan GALAXY observational study, ctDNA-based detection of molecular residual disease was predictive of survival outcomes and benefit of adjuvant chemotherapy in patients with resectable colorectal cancer.

In the phase 2 adaptive BELLINI trial, patients with early-stage triple-negative breast cancer received neoadjuvant nivolumab with or without ipilimumab, showing immune activation, clearance of circulating tumor DNA and promising clinical response rates, especially in patients preselected based on high levels of tumor-infiltrating lymphocytes.

In the observational SCRUM-Japan GOZILA study, after a median follow-up of 11 months, patients with metastatic gastrointestinal tumors who received biomarker-matched therapies based on circulating tumor DNA profiling showed a greater clinical benefit than those receiving unmatched therapy.

In the phase 2 NICHE-3 trial, patients with locally advanced mismatch repair-deficient colon cancer who were treated with neoadjuvant anti-PD1 and anti-LAG3 agents showed high rates of pathological responses, requiring validation in larger trials.

In the I-SPY2.2 trial, patients with high-risk stage 2/3 breast cancer received neoadjuvant datopotamab–deruxtecan, followed by sequential chemotherapy with or without targeted therapy, with the option of early surgical resection after each block of therapy. In a subgroup of patients, the sequential treatment strategy was superior to standard of care.

In the I-SPY2.2 trial, patients with high-risk stage 2/3 breast cancer received neoadjuvant datopotamab–deruxtecan plus durvalumab, followed by sequential chemotherapy with or without targeted therapy, with the option of early surgical resection after each block of therapy, showing that de-escalation of therapy is possible for several patient subgroups without compromising outcome and avoiding toxicity of standard chemotherapy.

A novel application of antisense oligonucleotide (ASO) technology, developed to treat a single patient, adds to the growing number of ‘personalized’ therapies for rare diseases; but pathways to implementation and access are urgently needed.

Post-approval evidence generation is essential for high-quality clinical care and should be a shared priority for clinicians, health systems, payors, and the medical products industry, as well as the FDA and federal agencies.

In the non-randomized phase 3b/4 DESTINY-Breast12 study, trastuzumab deruxtecan (T-DXd) treatment of patients with HER2⁺ advanced breast cancer and active or stable brain metastases showed consistent intracranial activity and systemic efficacy of T-DXd.

In people undergoing medical abortion after 12 weeks of pregnancy, those who took the first dose of misoprostol at home were less likely to require overnight hospitalization.