A blueprint to accelerate rare pediatric gene therapy approvals
Nature Medicine, Published online: 25 March 2026; doi:10.1038/s41591-025-04115-6
A blueprint to accelerate rare pediatric gene therapy approvalsNature Medicine, Published online: 25 March 2026; doi:10.1038/s41591-025-04115-6
A blueprint to accelerate rare pediatric gene therapy approvalsNature 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.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.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.Nature Medicine, Published online: 24 March 2026; doi:10.1038/s41591-026-04253-5
In a large UK-based primary care randomized trial, immediate artificial intelligence (AI)-driven prioritization of chest X-rays did not significantly shorten time to computed tomography or lung cancer diagnosis versus standard workflow, suggesting that AI-based prioritization alone is unlikely to accelerate the lung cancer diagnostic pathway.Nature Medicine, Published online: 16 March 2026; doi:10.1038/s41591-026-04306-9
In cohort 4 of the ILUSTRO trial, combination of anti-CLDN18.2 zolbetuximab plus mFOLFOX6 and nivolumab in patients with CLDN18.2-positive, HER2-negative metastatic gastric or gastroesophageal junction adenocarcinoma led to encouraging clinical efficacy, supporting the testing of this combination in a phase 3 trial.Nature Medicine, Published online: 12 March 2026; doi:10.1038/s41591-026-04265-1
In this phase 1, open-label dose-escalation study in healthy adults found that the mRNA vaccine (mRNA-1215), encoding the Nipah virus Malaysian strain chimeric pre-fusion F protein linked to glycoprotein G, was safe and induced elevated immune responses at 1 year of follow-up, indicating that this is a promising vaccine candidate for further development.Nature Medicine, Published online: 12 March 2026; doi:10.1038/s41591-026-04278-w
A real-world study showed that introducing a cognitive layer architecture to support specialized psychotherapeutic reasoning capabilities in general-purpose chatbots improved depression and anxiety symptoms compared to chatbots or therapists alone.Nature Medicine, Published online: 12 March 2026; doi:10.1038/s41591-026-04252-6
The authors propose a framework for clinical AI evaluation within simulated digital hospital environments that capture the evolving constraints, and cascading effects, of clinical decisions.Nature Medicine, Published online: 11 March 2026; doi:10.1038/s41591-026-04288-8
Author Correction: Global, regional, and national burden of chronic respiratory diseases and impact of the COVID-19 pandemic, 1990–2023: a Global Burden of Disease studyNature Medicine, Published online: 11 March 2026; doi:10.1038/s41591-026-04272-2
Elevated serum levels of phosphorylated tau are not specific to Alzheimer’s disease and may also serve as a diagnostic tool for the most common types of systemic amyloidosis, with potential utility in distinguishing amyloidosis-related polyneuropathy from polyneuropathy of other etiologies.Nature Medicine, Published online: 11 March 2026; doi:10.1038/s41591-026-04241-9
This Review highlights insights from recent clinical trials and discusses critical factors for optimizing cancer vaccines, with a focus on proxies for vaccine efficacy, neoantigen selection, modular platforms and early intervention.Nature Medicine, Published online: 09 March 2026; doi:10.1038/s41591-026-04239-3
In a prespecified ancillary analysis of the COSMOS randomized trial, supplementation with daily multivitamins, but not with cocoa extract, over the course of 2 years decreased biological aging, as measured by epigenetic aging clocks.Nature Medicine, Published online: 09 March 2026; doi:10.1038/s41591-026-04237-5
WHO STEPS survey data help assess global physical activity inequalities, and inform a new framework to recognize the impact of physical activity on health beyond cardiometabolic disease.