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A systematic review, combined with a stakeholder survey, presents an overview of current practices and recommendations for dataset curation in health, with specific focuses on data diversity and artificial intelligence-based applications.
Evidence that a dry-season outbreak of malaria in Dire Dawa, Ethiopia is caused by Anopheles stephensi carrying Plasmodium falciparum with diagnostic and drug resistance mutations calls for heightened vector surveillance in both urban and rural settings.
Comparison of representation of blind and deaf participants in the All of Us Research Program (AoURP) with CDC national estimates demonstrates that these populations are underrepresented in the AoURP cohort, raising concerns about the generalizability of findings generated using this dataset.
In the ongoing phase 1/2 BNT211-01 trial, CLDN6-specific chimeric antigen receptor (CAR)-T cells given with or without CARVac, a CAR-T cell-amplifying RNA vaccine, were well-tolerated and exhibited encouraging clinical activity in patients with relapsed or refractory CLDN6-positive solid tumors, with the highest response rate in patients with germ cell tumors.
Targeted depletion of TRBV9+ T cells induces remission in a single patient with ankylosing spondylitis, with significant improvements in functional and mobility metrics.
In a large multi-cancer cohort, a single liquid biopsy assay enables the detection of four epigenomic modifications, allowing the monitoring of expression of potential drug targets and resistance genes.
In a pilot feasibility study involving recipients of allogeneic hematopoietic stem cell transplants, adherence to a dietary intervention and its effects on the gut microbiota were assessed in the run-in to a study testing the effects of modulation of the microbiome on transplant outcomes.
The bispecific molecule tebotelimab, which blocks both PD-1 and LAG-3, is well tolerated as a monotherapy and in combination with the anti-HER-2 antibody margetuximab and elicits encouraging clinical activity in solid tumors with high LAG-3 levels and/or expression of IFN-γ-regulated genes.
In the phase 3 MAESTRO-NAFLD-1 trial, the liver-targeted thyroid hormone receptor-β selective agonist resmetirom was well tolerated and improved liver biomarkers in adults with nonalcoholic fatty liver disease.
In a retrospective analysis of patients with Lynch syndrome and primary cancers treated with immune checkpoint inhibitors, 12% developed subsequent malignancies, suggesting that this treatment may not eliminate risk in individuals predisposed to mismatch repair-deficient cancers, and ongoing surveillance is warranted.
In this pre-specified interim analysis, patients with resected stage IIB/C melanoma who received adjuvant nivolumab had significantly prolonged recurrence-free survival compared to placebo-treated patients, providing another treatment option for this population.
In the SURMOUNT-3 trial, once-weekly treatment with tirzepatide was demonstrated to result in clinically meaningful additional weight loss in adults with overweight or obesity following initial successful weight loss of at least 5% body weight with intensive lifestyle intervention.
In the first stage of the BR.36 adaptive trial in patients with non-small cell lung cancer receiving anti-PD1 immunnotherapy, the primary endpoint of concordance between circulating tumor DNA (ctDNA) molecular response and RECIST response was met. The results will inform the second, ctDNA-directed stage.
In the phase 3 STAR trial, subretinal administration of the AAV2-based gene therapy timrepigene emparvovec in patients with choroideremia did not lead to a significant difference in the primary endpoint of 15-letter Early Treatment Diabetic Retinopathy Study (ETDRS) improvement in visual acuity from baseline compared to untreated controls.
Microglia mediate the early and selective degeneration of corticostriatal synapses and the development of cognitive deficits in presymptomatic Huntington’s disease via the complement cascade.
Pre-exposure prophylaxis (PrEP) with daily antiretrovirals to prevent human immunodeficiency virus type 1 acquisition has not been shown to be consistently effective in cisgender women. Modeling adherence to daily PrEP clarifies how robust protection can be achieved.
Results from the DiViD Intervention, a phase 2 randomized, placebo-controlled trial, showed that antiviral treatment with pleconaril and ribavirin for 6 months resulted in higher endogenous insulin production in children and adolescents with new-onset type 1 diabetes.
In the first-in-human trial of elranatamab, patients with refractory or relapsed multiple myeloma who received the bispecific antibody against BCMA and CD3 experienced no dose-limiting toxicities during dose escalation, and the agent showed promising clinical efficacy.
In a phase 2 trial, the combination of gemcitabine, cisplatin and anti-PD-1 led to a clinical complete response in 43% of patients with muscle-invasive bladder cancer, which facilitated bladder sparing and was associated with long-term bladder-intact metastasis-free survival.
Reactivation of latent SIV with an IAP antagonist, coupled with immunotherapeutic approaches, reduced replication-competent SIV in lymph node CD4+ T cells in rhesus macaques. Further exploration targeting these pathways to reduce the HIV viral reservoir is warranted.