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Khabre desk
Recent reporting, analysis and explainers from the Clinical Research desk.
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A phase 2 trial in resectable non-small cell lung cancer found a major pathological response in 70.1% of patients who underwent surgery after perioperative ivonescimab plus chemotherapy. The study was noncomparative, and median event-free survival was not mature at the analysis cutoff.
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A six-month study of 356 perimenopausal women in China found that microbiome changes during hormone therapy differed by body site. Microbial patterns present before treatment were associated with differences in hormonal and symptom responses.
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A small phase 1b trial reported fewer bloodstream infections among donor stem-cell transplant recipients given SER-155 with vancomycin conditioning than among placebo recipients. The result is an early clinical signal from a small group, and the tested regimen combined both treatments.
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In the phase 2 PHERGain trial, a PET- and pathology-guided strategy had an estimated five-year invasive disease-free survival of 89.5% among patients assigned to the adaptive-treatment group who underwent surgery. Exploratory ctDNA analyses linked early clearance with higher pathological complete response rates and better five-year outcomes.
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A Nature Medicine study found that T-cell patterns in blood and cerebrospinal fluid were associated with ALS progression in different ways. CMV-specific immune responses were also linked to faster progression or shorter survival in some patient groups.
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A medRxiv preprint describes three machine-learning-derived autism profiles in two cohorts of autistic children. The profiles showed different associations with common and rare genetic variants, although the authors say larger-cohort validation is needed.
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A medRxiv analysis of 354 FDA oncology approvals from 2014 through early 2025 found surrogate endpoints had become the dominant primary measure used for approval. The authors also reported different relationships between surrogate-endpoint benefits and overall survival for immunotherapy and non-immunotherapy approvals.
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A medRxiv preprint describes a value-based care program for children and adolescents with type 1 diabetes at one US pediatric medical centre. From 2021 to 2025, reported HbA1c and acute-care measures improved, although the descriptive study cannot establish that the program caused the changes.
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A blinded preprint compared five large language models on 17 synthetic emergency-department cases. All named the reference diagnosis, but triage assignments and clinician ratings of response quality differed.
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A medRxiv preprint reports five genetic loci associated with age at Parkinson’s disease onset in a meta-analysis of 50,859 people with the disease. The findings include signals near SNCA, GBA1 and HIP1R, while the APOE association may reflect effects on longevity.
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In the phase 1/2 START-001 trial, four of 27 participants at selected doses had confirmed partial responses to invikafusp alfa. The early results also supported 0.08 mg/kg as the recommended phase 2 dose.
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In the randomized AMPLIFY-7P phase 2 trial, camupepimut did not significantly improve disease-free survival across all participants after pancreatic cancer surgery. Prespecified subgroup analyses and immune-response findings provide signals for testing in a larger trial.
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A medRxiv preprint comparing APOLLAI with emergency medical services raters on recordings from 295 patients with suspected stroke reports higher sensitivity for the AI system across five neurological tasks. The authors say prospective validation in real-world EMS conditions is needed.
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A single-centre preprint reports that disease-specific models using one plasma protein panel classified Alzheimer disease, Parkinson disease and ALS in a 302-person neurological cohort. The authors say multicentre external validation is needed before clinical application.
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A statistical model estimated that testing for recent tuberculosis infection could reduce the median number of people needed for a vaccine trial by 4.2- to 6-fold compared with long-lasting IGRA positivity under favourable conditions. The estimates are preprint model results, and the authors say candidate tests still need to be characterised.
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An analysis of All of Us health records estimated that about one in nine adults who had experienced Long COVID carried a corresponding diagnosis code, under assumptions comparing the records with federal survey data. Models rebuilt from released counts showed moderate ability to predict later coding, but performance varied by metric and site.
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A medRxiv preprint reports that surgically induced renovascular hypertension caused white-matter injury in rats, with brain AT1R signaling implicated in the damage. Human cohort analyses linked renal artery stenosis and angiotensin receptor blocker use with MRI measures of white-matter disease.
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A medRxiv preprint reports that trial eligibility criteria can create apparent differences in lipid-lowering treatment response by polygenic risk score, even when the true genetic interaction is set to zero. Its cohort analyses found little evidence that coronary artery disease risk scores modified statin effects.
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A randomised trial at BMJ Open found that asking authors to use a SPIRIT-based template during revision modestly improved the completeness of randomised trial protocols. Revision times were similar between groups, although some responding authors found the template time-consuming.
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A medRxiv preprint tested eight general-purpose large language models on 100 neurology board-style questions. Confidence estimates were often too high, while agreement across repeated answers best distinguished correct from incorrect responses.
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A BMJ obituary recounts Alan Lucas’s 1982 multicentre trial of feeding in preterm infants and later follow-up research on development and health. It credits Lucas with coining “nutritional programming” for the idea that early nutrition can shape outcomes later in life.
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A UK observational study followed 8,127 women receiving GLP-1-based treatment through a structured digital care programme. Among those with six-month weight measurements, average weight loss was 15.5% of starting weight.
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A single-site phase 1 trial evaluated D2C7-ATC, an EGFR-targeted antibody-toxin conjugate, in recurrent malignant glioma. Among 72 patients with IDH-wild-type recurrent glioblastoma, median overall survival was 7.9 months.
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A medRxiv preprint reports rare biallelic BECN1 variants in two unrelated families and connects the gene to AMPA-receptor regulation. Mouse and zebrafish experiments support the proposed biological mechanism.
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An Insilico Medicine preprint reports AUROCs of 0.828 for efficacy and 0.809 for safety/toxicity endpoint prediction on a test set of trials initiated in 2021 or later. It also introduces a benchmark of 2,040 ongoing trials for prospective evaluation as outcomes become available.
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A retrospective study using US health records found that dementia-risk associations varied among individual angiotensin receptor blockers (ARBs). The results are observational and the authors say randomized trials are needed for confirmation.
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In a phase 1a study of 20 healthy adults, 15 developed a fourfold rise in antibody titres against both cholera serotypes after a second PanChol dose. The preprint also reports reduced vaccine shedding after the booster, an indirect measure of intestinal immunity rather than a measure of protection from cholera illness.
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A cross-sectional study of 6,308 summary reports in the FDA’s MAUDE database found frequent reporting errors, missing device-problem detail and delays. The reports covered 159,503 adverse events from July 2019 to June 2025.
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A medRxiv preprint pooling two randomized trials found no statistically significant difference in 90-day mortality between intravenous cefazolin and antistaphylococcal penicillins for adults with MSSA bacteremia. Estimates for kidney injury and treatment discontinuation favoured cefazolin in direction but were imprecise.
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A medRxiv preprint reports that a fine-tuned ECG foundation model detected rheumatic mitral stenosis across 12-, 6- and 1-lead ECG configurations. Its external-validation AUROC ranged from 0.77 to 0.82, supporting further study of ECG screening and echocardiography triage.
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A single-centre retrospective study found routine clinical data could help rank likely genetic conditions in children with congenital heart disease. The AI models were evaluated in a selected cohort, so the results are a research finding rather than evidence of clinical diagnostic performance.
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A medRxiv preprint reports lower HbA1c in the BCG group of a randomized, placebo-controlled phase 2 trial, with follow-up extending to eight years. The abstract also reports reduced insulin dose and no recovery of C-peptide from negligible baseline levels.
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A medRxiv preprint reports five DNA-methylation clusters in 138 Wilms tumours and a classifier that identified WT1-driven tumours with about 90% accuracy. The researchers say using these patterns to guide genetic reanalysis uncovered previously unrecognised WT1 variants, including one that led to surveillance enrolment.
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In a study of 700 simulated conversations, five free AI chatbots maintained sleep apnoea referral recommendations in neutral exchanges but did so less often when the simulated patient minimised symptoms. The medRxiv preprint measured chatbot responses to scripted scenarios, not outcomes in real patients.
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An analysis of more than 300 million laboratory tests reconstructed group-level changes around the final menstrual period and found step-like shifts across many body systems. The study also found hormone changes beginning more than a decade before that point and an observational association between menopausal hormone therapy and smaller shifts.
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In a prospective study of 16 neurologically intact patients under propofol-based general anesthesia, norepinephrine raised mean arterial pressure by about 26% without increasing cerebral blood flow. The findings apply to this study setting and do not directly establish how pressure augmentation affects patients with acute neurological conditions.
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A prospective six-patient preprint evaluated Hiporfin for fluorescence-guided surgery and cavity photodynamic therapy in recurrent glioblastoma. Fluorescence had moderate specimen-level diagnostic accuracy, with some tumour samples not fluorescing.
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A Nature Medicine scoping review found that 482 of 3,967 studies citing TRIPOD or TRIPOD+AI reported sharing analytical code. Of 380 repositories the researchers could characterize, documentation, dependency details and tests were often missing.
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A 12-patient preprint reports that duplex sequencing detected the aristolochic acid-associated SBS22a mutation signature in blood and urine from patients with signature-positive urothelial tumours. The findings are a proof of concept, not yet a validated population screening test.
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A cross-sectional UK Biobank study found that white matter lesion volume had a different association with brain parenchymal fraction in people carrying certain NOTCH3 variants. The findings come from a medRxiv preprint and need to be interpreted in light of the small number of carriers studied.
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A secondary analysis of the two-year FINGER trial found that Alzheimer’s-related blood markers were associated with cognitive trajectories, but did not significantly modify the cognitive benefit of a multidomain lifestyle intervention. The findings come from a medRxiv preprint.
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A medRxiv preprint describes MINT, a foundation model trained and validated on 766,733 pediatric emergency visits. The authors report advantages over task-specific models in 21 of 25 comparisons and stronger performance than physicians on one respiratory-support forecasting task.
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A phase 1 follow-up of four patients with relapsed or refractory multiple myeloma found that one maintained a stringent complete response for 15 months. The other three progressed within five months, and median progression-free survival was 4.0 months.
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A medRxiv preprint reports that muFM, a model pretrained on more than 1.7 million unlabeled micro-ultrasound images, improved performance on three prostate image-analysis tasks. Gains were largest when the model was fine-tuned with limited annotated data.
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Researchers used self-reported data from 6.1 million people in 118 countries to create reference curves for pain across ages 5 to over 100. The estimates show different age patterns by pain site and higher late-life prevalence of several pain types in lower-HDI settings.
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A study of 62,560 individuals across diverse ancestries found no evidence that rare variants in TMEM106B are associated with Parkinson's disease risk. The findings suggest the gene's rare variation does not play a major role in PD susceptibility, despite evidence that TMEM106B expression is altered in the brains of people with the disease.
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A medRxiv preprint reports that three adapted machine-learning models were evaluated on videourodynamic data from an independent cohort of patients with spina bifida. A model combining pressure-volume measurements and fluoroscopic images had the strongest reported results for predicting hydronephrosis and classifying bladder dysfunction.
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A study of 30,533 US adults found that among three routine blood-test inflammatory indices, only the neutrophil-to-lymphocyte ratio independently predicted cardiovascular mortality, with SII and PLR adding little predictive value.
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A retrospective preprint describes a machine-learning model that combines CT-derived radiomics with clinical information to predict outcomes after spontaneous intracerebral haemorrhage. The authors report improved performance over clinical and conventional imaging variables alone.
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A cluster-randomized trial in northern Nigeria found higher card-verified measles vaccination coverage when small-quantity nutrient supplements were offered alongside routine immunization. The study tested a delivery strategy in 20 clusters, not the effect of the supplement alone.
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A Nature Medicine study reports that urine cell-free RNA sequencing detected localized bladder cancer with 95% sensitivity at 90% specificity. The researchers also investigated its potential for detecting residual disease and predicting response to bladder treatments.
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A medRxiv preprint reports that protein profiles from the esophageal String Test distinguished active eosinophilic esophagitis from healthy and reflux-disease groups. The exploratory findings point to a mix of inflammatory, epithelial and tissue-remodelling proteins.
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A medRxiv preprint reports that StrokeAgent matched expert-adjudicated final decisions in 86% of a curated set of 100 acute-stroke cases. The system processes clinical records, laboratory results, ECG waveforms and CT images as information becomes available.
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A medRxiv preprint reports that a time-aware machine-learning model identified some cases of hypoxic-ischaemic encephalopathy (HIE) hours before delivery using labour monitoring data. The analysis covered 174,186 deliveries and evaluated performance at a fixed 15% false-positive rate.
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A preprint meta-analysis of 14 observational studies found that preoperative anaemia was associated with higher odds of transfusion, kidney complications, early mortality and infection after adult cardiac surgery. The authors report low or very low certainty for these findings and say the observational evidence cannot establish causation.
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A prospective cohort study of 12,235 adults in England linked more days above WHO daily NO₂ guideline values with faster subsequent decline in memory and verbal fluency. The observational findings come from a medRxiv preprint.
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Researchers report open-source MATLAB and Python software that recovered ECG waveforms with zero error from non-clipped GE MUSE vector PDFs in a test of 1,632 PDF–XML pairs. The method uses information retained in the PDFs’ vector encoding and is specific to MUSE-generated files.
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A medRxiv preprint describes FRANK, a neural-network classifier trained on childhood tumour and normal-tissue RNA-seq data. The authors report 98.3% overall accuracy across nine validation datasets containing 16,398 samples.
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A small cross-sectional preprint reports overlapping clinical and immune findings among people with post-acute COVID-19 and a group with persistent symptoms following COVID-19 vaccination. The results describe similarities between the groups but do not determine what causes the symptoms.
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A medRxiv preprint reports different somatic mutation patterns in blood and arterial samples from patients undergoing coronary bypass surgery. Only four identical variants were found in both tissues among 221 matched pairs.
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A BMJ research methods paper presents INSPECT-SR, a 21-check tool for assessing the trustworthiness of randomised controlled trials in systematic reviews. It guides reviewers to judge concerns across four domains, but is not a fraud-detection or risk-of-bias tool.
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A medRxiv preprint reports higher C-index estimates for several survival outcomes when Evo2-derived tumor-sequence features were added to existing risk models. The study analysed 102 patients with soft-tissue sarcoma and a separate 129-patient TCGA cohort; clinical use requires further validation.
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A medRxiv preprint reports improved clinical outcomes and changes in immune markers after N-acetylcysteine treatment in cancer patients with severe COVID-19. The clinical comparison used severity-matched patients treated before the trial began.
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A systematic review and external validation assessed 48 models for predicting four adverse pregnancy outcomes using more than 1.58 million UK pregnancies. Most showed limited discrimination and substantial miscalibration in the UK cohort.
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An analysis of the NHS-Galleri trial reports Galleri’s performance across three annual screening rounds, including falling episode sensitivity in later rounds. The trial’s primary endpoint—a reduction in stage III/IV cancer diagnoses—was not met.
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A Nature Medicine study identified menopause-related blood protein patterns associated with hormonal changes, brain aging and less favourable cognitive outcomes later in life. The observational findings provide a research framework for studying how the menopause transition may intersect with dementia risk.
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A medRxiv preprint describes a method that distils multimorbidity reasoning from large language models into smaller models using synthetic cohorts shaped like UK Biobank data. The resulting models achieved Spearman correlations of 0.75–0.89 and produced scores associated with improved survival prediction in a UK Biobank analysis.
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A six-month pilot in 138 cognitively normal adults used family history, genetics and plasma pTau217 to identify people at higher Alzheimer’s risk. Participants who received personalised lifestyle coaching reported less worry, while cognitive scores and lifestyle-risk measures remained unchanged and pTau217 showed only a slower rise.
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A medRxiv preprint describes an open-source framework that synchronized 6,384 hours of chronic deep brain stimulation recordings with wearable data from three people with obsessive-compulsive disorder.
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A medRxiv preprint analysed 344 postoperative drain-fluid samples from 67 pancreatic and colorectal surgery patients. It found that leakage was associated with different protein patterns over time in the two types of surgery.
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A retrospective study of 10,634 percutaneous coronary interventions found radiation-alert threshold breaches in 0.93% of procedures and 2.1% of complex procedures. The authors developed an internally assessed score using early clinical and procedural variables.
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A medRxiv preprint reports that Ataraxis Breast CTX, a multimodal AI model using clinical data and H&E images, separated five-year disease-free survival in a selected group of 556 clinically high-risk, node-positive HR+/HER2- early breast cancer patients. An exploratory analysis found that everolimus benefit varied by the model’s score.
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A medRxiv preprint reports that chronic brain recordings from 24 people receiving deep brain stimulation for treatment-resistant OCD distinguished likely responders before stimulation and tracked early treatment engagement. The findings describe a potential biomarker framework, but the study is still a preprint.
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An ETH Zurich preprint used physiological and environmental signals from 20 participants to distinguish ambient temperature, humidity and physical exertion with 94.7% Random Forest accuracy. The exploratory study is an early step toward person-centred heat monitoring and requires testing in real-world settings.
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A medRxiv preprint analysing 8,741 female participants older than 70 reports that associations between menopause hormone therapy, cognition and brain structure differed by treatment formulation and APOE ε4 status. Among APOE ε4 carriers, estradiol-based therapy was linked to more favourable cognitive and imaging measures than conjugated equine estrogens.
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A medRxiv preprint found that appropriate-use guidance reduced nuclear stress-test ordering for some emergency observation-unit patients. Echocardiogram use increased, leaving combined use of the two tests essentially unchanged.
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A medRxiv preprint links moderate-to-vigorous leisure-time activity with lower BMI, waist-to-height ratio and central-adiposity risk in adults aged 60 to 80 at high cardiovascular risk. The analysis found the strongest reduction in central-adiposity risk up to about 400 MET·min/day.
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A medRxiv protocol describes the MINTS study, which will examine 14 home-based motor-imagery neurofeedback sessions in 21 people with chronic stroke and persistent upper-limb impairment. The study will combine mobile EEG, stationary EEG, functional MRI and repeated motor assessments.
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A medRxiv preprint reports 41 genetic risk regions associated with juvenile idiopathic arthritis, including 14 not previously reported in the analysis. The study also describes a polygenic risk score and possible mechanisms involving HLA-A*02:01, ERAP2 and DNA topology.
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A medRxiv preprint reports that laromestrocel reduced measures of brain inflammation and atrophy in 49 people with mild Alzheimer’s disease. The findings come from an early clinical study and have not yet been published as a peer-reviewed journal article.
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A medRxiv preprint evaluated 14 general-purpose large language models on eight schizophrenia symptoms in clinical summaries from a 704-person severe mental illness cohort. Performance was poor to moderate overall, with negative symptoms extracted less accurately than positive symptoms.
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A medRxiv preprint reports that plasma p-tau217 showed the broadest association with later Alzheimer’s-related brain atrophy among cognitively unimpaired participants. The observational analysis included 818 people and 2,293 longitudinal brain MRI scans.
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A medRxiv preprint describes a modified endpoint QuIC assay that detected prion seeding activity in sporadic CJD brain samples within six hours, with some results available in two hours. The assay showed 100% sensitivity and specificity in the reported CSF testing.
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A 2026 readjudication of the 330-person ADVOCATE trial found avacopan produced remission rates similar to a prednisone taper in granulomatosis with polyangiitis and microscopic polyangiitis. The preprint also reports lower glucocorticoid exposure and toxicity with avacopan.
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A medRxiv preprint used proteome-wide Mendelian randomization to identify 11 plasma proteins associated with early-onset stroke, defined as stroke occurring before age 60. The strongest signals involved coagulation and thromboinflammatory pathways, with Factor XI highlighted as a potential therapeutic target.
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A prospective medRxiv study of 121 adults found six anaerobic species enriched in anal squamous cell carcinoma compared with high-grade lesions. The microbial pattern persisted during follow-up and changed after treatment initiation.
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A medRxiv preprint reports that 12 weeks of adapted tango improved clinical and motor outcomes in people with mild-to-moderate Parkinson’s disease. Leader and follower roles were associated with partly different changes in brain-network connectivity.
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An observational study of people with relapsing-remitting multiple sclerosis found Epstein–Barr virus-associated immune signals and expansion of atypical B cells weeks to months before clinical relapse. The findings identify a possible peripheral precursor of relapse, but the connection to inflammation in the brain and spinal cord remains unresolved.
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A single-centre observational preprint reports treatment-associated improvement in several catatonic signs among autistic patients receiving electroconvulsive therapy. The findings highlight how catatonia may resemble worsening autism during later regression.
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A six-patient preprint found higher interferon-related gene scores in blood samples associated with dermatomyositis flares. The feasibility study used weekly at-home blood microsampling and RNA sequencing.
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A medRxiv preprint analysing preoperative imaging from 999 people with newly diagnosed glioblastoma found that a tractography-based measure of tumour involvement across white-matter pathways was more consistently associated with overall survival than tumour volume. The finding is based on a multi-institution imaging and survival analysis, not a treatment trial.
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A medRxiv preprint analyses more than 4.29 million audiograms and models human hearing as a continuous five-dimensional system rather than a set of fixed types. The secondary analysis could provide a new framework for studying hearing change, but it is not a clinical trial or treatment study.
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A medRxiv preprint analysing 50,463 free-living meals from 992 adults without diagnosed diabetes found that model-derived glucose-response scores estimated from three days of monitoring approximated scores from 14 days. The analysis also identified two major dimensions of individual meal responses and tested them against 4,524 held-out standardized meals.
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A Nature Medicine study describes a fully on-premise clinical AI agent that combines diagnostic reasoning with repeated-run consistency checks. On a seven-disease benchmark, the system retained 98.9% accuracy among cases selected for autonomous handling at a defined consistency threshold, while routing the rest for review.
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A medRxiv preprint reports that altered placental LBX1 was associated with spontaneous preterm birth in analyses of 70 placentae and 53 maternal plasma samples. Cell findings suggest a pathway involving nuclear import, ribosomal disruption and the unfolded protein response.
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A medRxiv preprint compared matched blood and bone-marrow cell-free DNA from 74 people with multiple myeloma. Peripheral-blood cfDNA matched or exceeded marrow-plasma cfDNA for several genomic signals, while fragment patterns varied by sample compartment and handling.
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A medRxiv preprint analysing 12,944 participants from nationally representative cohorts in China, England and the United States found that higher estimated cardiorespiratory fitness was associated with a lower risk of developing two or more cardiometabolic conditions. The observational study suggests that a non-exercise fitness estimate may help characterise risk, but it does not establish that increasing fitness prevents these conditions.
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A medRxiv preprint reports seven genomic risk loci associated with myalgic encephalomyelitis/chronic fatigue syndrome after analysis of UK Biobank and All of Us data. The findings provide leads for studying the condition’s biology, but the study is based on a preprint and no locus was significant across all three cohorts.
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A medRxiv systematic review and meta-analysis found that the blood biomarker GFAP distinguished intracerebral hemorrhage from ischemic stroke, with pooled specificity of 87.2%. Accuracy was higher in samples taken within two hours, but sensitivity remained limited, particularly for smaller hemorrhages.
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A systematic review of 230 human low-intensity focused ultrasound experiments found that acoustic settings associated with neural facilitation or suppression differed between cortical and subcortical targets. The medRxiv preprint introduces SonoMap, an interactive resource for comparing these parameter-effect patterns.
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A medRxiv preprint combining genetic and single-cell analyses identifies bone-marrow macrophages as a possible link between aging and osteoporosis. It nominates TGFB1 and HNRNPUL1 as regulators in this relationship, based on observational, genetic and transcriptomic evidence.
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A medRxiv preprint evaluated a large-language-model pipeline for assessing risk of bias in neurological prognosis studies. Agreement with original human assessments was limited, while a small comparison suggested a similar level of agreement to that between human raters.
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A medRxiv preprint identifies two auditory nerve response patterns in older adults, including one associated with MRI and histological signs of reduced myelin integrity. People with this poorer-synchrony pattern reported greater hearing difficulties relative to their hearing thresholds.
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A medRxiv preprint evaluates BanffNET indices that summarise kidney-transplant biopsy injury and distinguish several Banff pathology categories. The model was assessed across 6,407 biopsies and a multi-reader study involving 67 pathologists.
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A medRxiv preprint reports that a risk score based on 28 gut microbial species was associated with cancer diagnoses during up to 10 years of follow-up. The finding comes from an observational analysis and represents a candidate biomarker for future validation, not a clinical screening test.
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A Nature Medicine study reports CRISP, a clinically oriented pathology foundation model developed using more than 100,000 frozen sections, with prospective evaluation in more than 3,000 patients.