Trained and validated on multimodal data from 14.5 million images from multicountry datasets, a foundation model is shown to increase diagnostic and referral accuracy of clinicians when used as an assistant in a trial involving 16 ophthalmologists and 668 patients.

Asking patients about their symptoms, functioning and well-being during clinical trials can influence trial outcomes, regulatory decisions and clinical practice.

Genetic background, plasma metabolome and dietary data from 4,215 women and 1,490 men reveal 57 metabolites whose associations with dementia risk vary by APOE4 genotype and other risk variants.

A report from the first 2,000 newborns enrolled in the Early Check program, which offered genome screening covering 198 genes associated with early-onset diseases, shows the feasibility and potential clinical utility of the approach, leading to 28 true positive results.

In a phase 3 trial for Huntington’s disease, pridopidine did not meet its primary endpoints but was safe and favored in some prespecified subgroup analyses.

In a phase 1/2a umbrella trial in patients with glioblastoma matching targeted therapies according to tumor molecular profiling, temsirolimus plus radiotherapy improved PFS compared to standard of care in patients with phospho-mTOR activation.

In this single-arm phase 2 trial in patients with HR⁺HER2⁻ advanced breast cancer, treatment with the HER3-targeting antibody–drug conjugate paritumab deruxtecan led to encouraging objective response rates, and comprehensive exploratory analyses indicate potential biomarkers of response.

In the secondary endpoint analysis of the phase 3 SONIA trial, circulating tumor DNA (ctDNA) analysis showed that a pretreatment aneuploidy-based high ctDNA level in patients with advanced breast cancer was associated with benefit from first-line treatment with CDK4/6 inhibitors.

A combination of mouse model data and data from a randomized phase 2 trial in patients with breast cancer in remission demonstrates feasibility, safety and a reduction in residual tumor cells following treatment with hydroxychloroquine and/or everolimus.

A digital twin, or virtual organ, can help clinicians and patients make better decisions, and can even help in the design of more-efficient clinical trials.

Luis Diaz describes how a molecular blood assay revealed the tiniest traces of DNA from a tumor, changing how we monitor cancer.

A study of over 1.2 million children found no links between aluminum-adjuvanted childhood vaccines and over 50 chronic diseases, including neurological, autoimmune and allergic conditions.

The recent defunding of USAID could lead to more than 14 million deaths, mostly in low- and middle-income countries, including 4.5 million child deaths — according to a study combining retrospective and forecasting analyses.

Genomic sequencing promises targeted treatment for every patient with cancer; the reality is much tougher to deliver.