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Consensus framework for the validation of generative AI: call for collaborators on the Validation Accords

Nature Medicine, Published online: 09 September 2026; doi:10.1038/s41591-026-04647-5

Consensus framework for the validation of generative AI: call for collaborators on the Validation Accords

Inhaled siRNA therapy targeting RAGE for pulmonary inflammation: a first-in-human randomized trial

Nature Medicine, Published online: 09 September 2026; doi:10.1038/s41591-026-04607-z

Following preclinical development, a randomized first-in-human trial showed that treatment with an inhaled, lung-epithelium-targeted siRNA directed against the receptor for advanced glycation end-products (RAGE) was safe and well tolerated and reduced RAGE levels in serum and bronchoalveolar lavage fluid.

Myeloperoxidase inhibition with mitiperstat in heart failure with preserved or mildly reduced ejection fraction: a randomized phase 2b trial

Nature Medicine, Published online: 09 September 2026; doi:10.1038/s41591-026-04615-z

In a phase 2 randomized clinical trial, treatment with the myeloperoxidase (MPO) inhibitor mitiperstat, intended to target the neutrophil–MPO inflammatory pathway, did not improve symptoms or exercise function in individuals with heart failure with preserved or mildly reduced ejection fraction.

From algorithms to patient outcomes — lessons from one of the first randomized trials of AI in medicine

7 September 2026 at 08:00

Nature Medicine, Published online: 07 September 2026; doi:10.1038/s41591-026-04633-x

The first generation of medical artificial intelligence (AI) was judged on whether algorithms could match clinicians. The next generation should be judged on whether carefully designed human–AI systems can improve patient outcomes.
Received — 27 May 2026 ⏭ Nature Medicine

Enabling secure discovery in trusted research environments with improved tooling

Nature Medicine, Published online: 26 May 2026; doi:10.1038/s41591-026-04401-x

Biobanks are introducing trusted research environments to enable secure data access, yet they can increase costs and complexity for biomedical researchers who use them. We discuss challenges and solutions and introduce the Python Phenofhy community tool for Our Future Health.

Fibroblast growth factor receptor inhibition for succinate dehydrogenase-deficient gastrointestinal stromal tumors: a phase 2 trial

Nature Medicine, Published online: 26 May 2026; doi:10.1038/s41591-026-04376-9

In a multicenter phase 2 trial, the fibroblast growth factor receptor inhibitor rogaratinib showed encouraging clinical efficacy in patients with succinate dehydrogenase-deficient gastrointestinal stromal tumors, suggesting a potential new treatment option for this patient population and demonstrating that an epigenetic mechanism of oncogene activation can be successfully targeted with a tyrosine kinase inhibitor.
  • ✇Nature Medicine
  • A rare case of gene therapy-linked cancer Karen O’Leary
    Nature Medicine, Published online: 22 May 2026; doi:10.1038/d41591-026-00027-1A rare case of genomic integration of an adenovirus vector led to a child developing a tumor — successfully removed by surgery — which highlights the importance of long-term safety monitoring in patients who receive gene therapy.
     

Semaglutide versus placebo in individuals with poor weight loss after bariatric surgery: a double-blinded, randomized, placebo-controlled trial

Nature Medicine, Published online: 22 May 2026; doi:10.1038/s41591-026-04416-4

At week 68, in patients who experienced poor weight loss following bariatric surgery, semaglutide was associated with 18.0% weight loss compared to 0.4% weight gain in patients receiving placebo.

AI-induced never-skilling in medical education

Nature Medicine, Published online: 22 May 2026; doi:10.1038/s41591-026-04438-y

Will medical trainees who rely on AI fail to develop foundational independent clinical reasoning? This Perspective outlines a precautionary framework to preserve foundational competence while supporting safe and effective AI integration in medical training.
Received — 8 April 2026 ⏭ Nature Medicine

Zodasiran for cholesterol and triglyceride lowering in patients with hyperlipidemia: final report of phase 1 basket trial

Nature Medicine, Published online: 07 April 2026; doi:10.1038/s41591-026-04307-8

In a phase 1 basket trial, the small interfering RNA zodasiran, targeting ANGPTL3, lowered triglycerides in patients with severe hypertriglyceridemia and lowered both triglycerides and low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolemia.

Isatuximab, carfilzomib, lenalidomide and dexamethasone in newly diagnosed multiple myeloma: a randomized phase 3 trial

Nature Medicine, Published online: 06 April 2026; doi:10.1038/s41591-026-04282-0

In the phase 3 EMN24 IsKia trial, transplant-eligible patients with newly diagnosed multiple myeloma who received isatuximab with carfilzomib, lenalidomide and dexamethasone pretransplant induction and post-transplant consolidation showed higher rates of measurable residual disease negativity after consolidation than patients who received carfilzomib, lenalidomide and dexamethasone.
Received — 4 April 2026 ⏭ Nature Medicine

Evidence of monkeypox virus clade IIb lineage A.2.2 in the Republic of the Congo and co-circulation of clade Ia, Ib and clade IIb

Nature Medicine, Published online: 03 April 2026; doi:10.1038/s41591-026-04256-2

Whole-genome phylogenetic analyses have identified a case of monkeypox virus clade IIb lineage in the Republic of the Congo and co-circulation of three clade lineages, emphasizing the importance of improved surveillance given the risk of possible recombination events in the future.
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