❌

Normal view

Levetiracetam therapeutically targets GABAergic synapses in diffuse midline glioma

Nature Medicine, Published online: 17 September 2026; doi:10.1038/s41591-026-04646-6

Results of this study show in experimental models and data from patient cohorts that the antiseizure medication levetiracetam is associated with longer survival and reduced tumor growth in diffuse midline glioma, but not hemispheric high-grade glioma, by selectively dampening GABAergic synaptic signaling, independently of its canonical SV2A-mediated primary antiseizure mechanism.

EBV reactivation priming of the peripheral immune system in multiple sclerosis relapse

Nature Medicine, Published online: 16 September 2026; doi:10.1038/s41591-026-04665-3

Increased expression of EBV reactivation genes in B cells and MS risk genes targeted by the EBV protein EBNA-2 precedes MS attacks, linking EBV reactivation and genetic risk to the development of MS relapses.

Liquid biopsy for early detection of pancreatic ductal adenocarcinoma

Nature Medicine, Published online: 16 September 2026; doi:10.1038/s41591-026-04625-x

In a prospective study involving 1,785 individuals from four countries, the PANXEON exosome-based biomarker, combined with carbohydrate antigen 19-9 levels, achieves high sensitivity for the detection of early-stage pancreatic cancer.

Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial

Nature Medicine, Published online: 16 September 2026; doi:10.1038/s41591-026-04636-8

In this single-arm phase 1 trial, an AAV gene therapy carrying the Padua variant of factor IX was well tolerated in 11 adolescents with hemophilia B and led to reductions in annualized bleeding rate.

Sex-specific biological aging clocks across organs and omics

Nature Medicine, Published online: 16 September 2026; doi:10.1038/s41591-026-04662-6

Sex-specific biological aging clocks across multiple organs and molecular systems show that female and male aging patterns can differ in organ-specific, disease-relevant ways.

Beyond the breakthrough for RAS-driven cancers

15 September 2026 at 08:00

Nature Medicine, Published online: 15 September 2026; doi:10.1038/s41591-026-04610-4

Two early clinical studies of selective KRASG12D inhibitors show encouraging activity in advanced cancers with G12D mutations, signaling a transformative change in the treatment landscape for RAS-driven cancers with potential applications across tumor types and stages of disease.

On-premise medical AI agents for reliable clinical decision-making

Nature Medicine, Published online: 15 September 2026; doi:10.1038/s41591-026-04609-x

An autonomous clinical AI agent enhances decision-making through on-premise deployment and reliability metrics, achieving high diagnostic accuracy and selective autonomy.

Broadly neutralizing antibodies in adult males living with HIV undergoing analytical treatment interruption: secondary and exploratory outcomes of the phase II randomized controlled RIO trial

Nature Medicine, Published online: 15 September 2026; doi:10.1038/s41591-026-04644-8

In the phase 2 RIO trial, there was delayed viral rebound and resistance to broadly neutralizing antibodies 3BNC117-LS and 10-1074-LS in adult males living with HIV undergoing analytical treatment interruption, and initial reservoir sensitivity to autologous antibodies was associated with a longer time to rebound.
  • ✇Nature Medicine
  • De novo mutations bridge parental reproductive factors and offspring health
    Nature Medicine, Published online: 14 September 2026; doi:10.1038/s41591-026-04676-0Whole-genome sequencing of 7,851 parent–offspring families enabled the identification of parent-of-origin and post-zygotic de novo mutations that were associated with distinct procedures of assisted reproductive technology (ART), independently of parental age at conception. Increased paternal mutational burden statistically mediates the effects of advanced parental age and ART on gestational duration and other bi
     

De novo mutations bridge parental reproductive factors and offspring health

14 September 2026 at 08:00

Nature Medicine, Published online: 14 September 2026; doi:10.1038/s41591-026-04676-0

Whole-genome sequencing of 7,851 parent–offspring families enabled the identification of parent-of-origin and post-zygotic de novo mutations that were associated with distinct procedures of assisted reproductive technology (ART), independently of parental age at conception. Increased paternal mutational burden statistically mediates the effects of advanced parental age and ART on gestational duration and other birth outcomes.

Rethinking the global health system for a new international order

Nature Medicine, Published online: 14 September 2026; doi:10.1038/s41591-026-04622-0

In this Comment, we argue for a global health system that recognizes and supports countries as the primary agents of action, oriented around global public goods and collective action, and served by a leaner set of global and regional institutions. We present a potential vision for the functions and distribution of labor in the system to inform ongoing reform discussions and draw critique, dissent and amendment.

Prospective evidence for conversational medical AI is hard, but non-negotiable

Nature Medicine, Published online: 14 September 2026; doi:10.1038/s41591-026-04639-5

Trust in clinical artificial intelligence (AI) cannot be benchmarked into existence. It must be earned through rigorous prospective studies in real-world clinical settings, where the hardest lessons often concern the humans and systems around the AI, not the technology itself.

Clinical usability of an explainable AI decision support tool and evaluation of multimodal models in NSCLC

Nature Medicine, Published online: 13 September 2026; doi:10.1038/s41591-026-04488-2

In a large international real-world study of non-small cell lung cancer, a multimodal explainable AI model outperformed established biomarkers for immunotherapy outcome prediction and improved physician decision-making.

A global digital navigator of human health for precision medicine

Nature Medicine, Published online: 11 September 2026; doi:10.1038/s41591-026-04621-1

The International Consortium of Digital Twins in Healthcare and Medicine was established to advance medical digital twin technology as a new infrastructure for precision health.

Teclistamab versus lenalidomide-dexamethasone in high-risk smoldering multiple myeloma: a randomized phase 2 trial

Nature Medicine, Published online: 11 September 2026; doi:10.1038/s41591-026-04642-w

In the randomized phase 2 ImmunoPRISM trial, patients with high-risk smoldering multiple myeloma (MM) showed higher rates of complete clinical responses in response to treatment with teclistamab compared with lenalidomide–dexamethasone, although longer follow-up is required to determine durable prevention of progression to MM.

Joint impact of pathological burden and cognitive resilience on Alzheimer’s disease risk

Nature Medicine, Published online: 11 September 2026; doi:10.1038/s41591-026-04635-9

A 15-year cohort study shows that Alzheimer’s dementia risk is jointly shaped by Alzheimer’s pathology and cognitive resilience, with high resilience linked to lower risk, even under greater pathology.

A clinically-oriented foundation model for intraoperative pathology

Nature Medicine, Published online: 10 September 2026; doi:10.1038/s41591-026-04703-0

CRISP, a vision-based pathology foundation model developed exclusively from frozen section slides, supports treatment decision-making throughout the surgical workflow with superior performance to current foundation models and extensive validation, including in a prospective cohort.

Computable longitudinal patient journeys from structured and unstructured EHR data

Nature Medicine, Published online: 10 September 2026; doi:10.1038/s41591-026-04695-x

A suite of large pre-trained language models accurately extracts computable clinical data from unstructured electronic health records and integrates the findings into knowledge graphs that can facilitate understanding patient trajectories and treatment responses in the real world.

Our Future Health and the next generation of population medicine

10 September 2026 at 08:00

Nature Medicine, Published online: 10 September 2026; doi:10.1038/s41591-026-04596-z

An early assessment of the UK’s Our Future Health project shows how national-scale cohorts may reshape population medicine by accelerating discovery and clinical recruitment, provided that selection and phenotyping biases are carefully measured and managed.
❌