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In a phase 2 randomized clinical trial, an inhibitor of cholesteryl ester transfer protein (CETP), obicetrapib, lowered low-density lipoprotein cholesterol when administered in conjunction with high-intensity statins, paving the way for studies investigating the effects of obicetrapib on cardiovascular events.
Single-cell genomic and transcriptomic analyses of longitudinal samples of patients with Richter syndrome reveal the presence and dynamics of clones driving transformation from chronic lymphocytic leukemia years before clinical manifestation
A comprehensive comparison of Alzheimer’s disease blood biomarkers in cognitively unimpaired individuals reveals that plasma p-tau231 and p-tau217 capture very early Aβ changes, showing promise as markers to enrich a preclinical population for Alzheimer’s disease clinical trials
Findings from the TUXEDO-1 trial demonstrate efficacy of the antibody–drug conjugate trastuzumab deruxtecan for treatment of brain metastases in patients with HER2-positive breast cancer.
The SIGNAL Phase 2 study of pepinemab immunotherapy in early Huntington’s disease (HD) did not meet its coprimary clinical efficacy endpoints, but had a favorable safety profile and showed a significant treatment-related reduction in caudate brain atrophy and reversal of the characteristic decline in brain metabolic activity that is typical of HD progression.
An engineered protein engages the efferocytosis pathway to induce amyloid-β engulfment, resulting in behavioral rescue in Alzheimer’s disease mouse models without the increased inflammation or vascular pathology associated with conventional antibody therapy
Preliminary results from a phase 1/2 trial with 18-month follow-up show that transplantation of CRISPR–Cas9 BCL11A-edited autologous hematopoietic cells in two children with β-thalassemia was safe and achieved transfusion independence.
In a phase 2 trial, monitoring tumor resistance mutations in blood using liquid biopsies allowed rationally guided subsequent therapy with anti-EGFR antibodies in patients with colorectal cancer.
To overcome limitations of previous genome-wide association studies of coronary artery disease, this study incorporates a cohort of individuals containing large fractions of Black and Hispanic individuals to provide a wider view of the genetic landscape of this disease.
A retrospective analysis of primary care records in the United Kingdom reveals individual symptoms associated with SARS-CoV-2 infections, which persisted for 12 weeks or more after infection, as well as risk factors associated with developing long COVID.
A report from large cohorts of adult survivors of childhood cancer demonstrates that genetic risk scores improve the risk prediction of developing severe obesity, providing opportunities for surveillance and mitigation interventions
Results from a pivotal single-arm phase 2 trial show that the repeated intratumoral administration of the oncolytic herpes virus G47∆ in residual or recurrent glioblastoma exhibits survival benefit and a safe profile.
Prospective evaluation of a machine learning-based early warning system for sepsis, deployed at five hospitals, showed that healthcare providers interacted with the system at a high rate and that this interaction was associated with faster antibiotic ordering.
Prospective evaluation of a machine learning-based early warning system for sepsis, deployed at five hospitals, showed that interaction of health-care providers with the system was associated with better patient outcomes, including reduced in-hospital mortality.
A new modeling study demonstrates that most countries could achieve 80-80-80 hypertension targets by 2040, promoting increased global equality for cardiovascular diseases with the greatest mortality benefits being seen in low-income countries.
An analysis of blood samples from longitudinal cohorts reveals insights on the dynamics of clonal hematopoiesis of indeterminate potential and proposes a model that could be used for individualized clone monitoring over time.
A large-scale computational analysis of over 40,000 patients with cancer who have detailed mutation profiles, treatment sequences and outcomes derived from electronic health records provides insights into a rich resource that will help to enable precision oncology.
Genomic characterization of the SARS-CoV-2 Omicron lineages BA.4 and BA.5, responsible for the fifth COVID-19 pandemic wave in South Africa, shows continued viral diversification and provides insights into the potential mechanisms underlying the ability of the new lineages to outcompete their predecessors.
An ecological analysis of 326 cities in 9 countries across Latin America found that changes in ambient temperature have a substantial contribution to all-cause mortality, with small increases in extreme heat associated with steep increases in mortality risk.
Analysis of the first sequences from the 2022 multi-country outbreak of monkeypox virus shows relatedness and substantial divergence from a 2018–2019 outbreak, suggesting rapid virus evolution with possible implications for human-to-human transmission.