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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
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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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Target product profiles for treatments to delay or prevent symptomatic Alzheimer’s disease

Nature Medicine, Published online: 03 April 2026; doi:10.1038/s41591-026-04305-w

To accelerate therapeutic development and equip stakeholders with clear benchmarks, the authors outline target product profiles for therapies designed to delay or prevent the onset of clinical symptoms of Alzheimer’s disease.
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Semaglutide on liver fibrosis and heart outcomes in patients at high risk of liver fibrosis: a prespecified analysis of the SELECT randomized trial

Nature Medicine, Published online: 02 April 2026; doi:10.1038/s41591-026-04281-1

A prespecified analysis from the SELECT trial showed that semaglutide reduces major adverse cardiovascular events by 20% compared with placebo, particularly in patients at high risk of fibrosis, as indicated by the Fibrosis-4 index.
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A deep joint-learning proteomics model for diagnosis of six conditions associated with dementia

Nature Medicine, Published online: 31 March 2026; doi:10.1038/s41591-026-04303-y

ProtAIDe-Dx is a deep joint-learning model that uses plasma proteomics to provide simultaneous probabilistic diagnoses across six conditions associated with aging.
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Benralizumab versus placebo for hypereosinophilic syndrome: a randomized, placebo-controlled phase 3 trial

Nature Medicine, Published online: 31 March 2026; doi:10.1038/s41591-026-04315-8

Benralizumab (an anti-IL-5 receptor α antibody), compared to placebo, significantly reduced the risk of first flare in patients with hypereosinophilic syndrome.
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In vivo generation of anti-BCMA CAR-T cells in relapsed or refractory multiple myeloma: a phase 1 study

Nature Medicine, Published online: 25 March 2026; doi:10.1038/s41591-026-04244-6

In a phase 1 trial, the in vivo generation of anti-BCMA CAR-T cells by lentiviral delivery was feasible and did not lead to dose-limiting toxicities in five patients with relapsed or refractory multiple myeloma.
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Pembrolizumab and olaparib in homologous-recombination-deficient metastatic pancreatic cancer: the phase 2 POLAR trial

Nature Medicine, Published online: 25 March 2026; doi:10.1038/s41591-026-04299-5

Results of the phase 2 POLAR trial show that biomarker-guided treatment in patients with metastatic pancreatic cancer based on homologous repair deficiency leads to encouraging clinical response rates in immune cell-infiltrated tumors.
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<i>LRRK2</i>-targeting antisense oligonucleotide in Parkinson’s disease: a phase 1 randomized controlled trial

Nature Medicine, Published online: 24 March 2026; doi:10.1038/s41591-026-04262-4

The first-in-human clinical trial of the LRRK2-targeting antisense oligonucleotide BIIB094 in Parkinson’s disease demonstrates that the treatment is well tolerated and produces dose-dependent reductions in cerebrospinal fluid levels of LRRK2 and phosphorylated Rab10, indicating successful target engagement.
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