An iPSC-derived neural progenitor cell therapy for subacute spinal cord injury: a phase 1 trial with long-term follow-up - Nature Medicine

A phase 1 trial evaluating the safety of iPSC-derived neural stem cell transplantation in subacute complete spinal cord injury, with 2–4 years of follow-up including MRI, neurological and functional assessments met its primary outcome.

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Longitudinal plasma proteomics predict phenoconversion to clinically manifest ALS - Nature Medicine

Longitudinal proteomic analysis of plasma from unaffected carriers of ALS-associated pathogenic variants revealed early protein changes before ALS symptoms and enabled mapping of their trajectories as well as prediction of the timing of phenoconversion

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An iPSC-derived neural progenitor cell therapy for subacute spinal cord injury: a phase 1 trial with long-term follow-up - Nature Medicine

A phase 1 trial evaluating the safety of iPSC-derived neural stem cell transplantation in subacute complete spinal cord injury, with 2–4 years of follow-up including MRI, neurological and functional assessments met its primary outcome.

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The genetic architecture of fibromyalgia across 2.5 million individuals - Nature Medicine

A multi-ancestry genome-wide association study meta-analysis across 2,563,755 individuals identifies 26 risk loci for fibromyalgia, including HTT, and finds genetic correlations with chronic pain and psychiatric and somatic disorders.

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A neuroprosthesis for restoring hand movement and sensation in a person with complete tetraplegia - Nature Medicine

A bidirectional sensorimotor neuroprosthetic system that decodes brain signals associated with movement intention while delivering patterned neuromodulation to the spinal cord and cortex provides long-term recovery of hand motor and sensory function, even when the device is off.

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Longitudinal plasma proteomics predict phenoconversion to clinically manifest ALS - Nature Medicine

Longitudinal proteomic analysis of plasma from unaffected carriers of ALS-associated pathogenic variants revealed early protein changes before ALS symptoms and enabled mapping of their trajectories as well as prediction of the timing of phenoconversion

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Lassa fever countermeasures gather pace - Nature Medicine

Two early phase clinical trials represent progress in Lassa fever vaccine and therapeutic development, including the first interventional treatment trial in decades.

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An iPSC-derived neural progenitor cell therapy for subacute spinal cord injury: a phase 1 trial with long-term follow-up - Nature Medicine

A phase 1 trial evaluating the safety of iPSC-derived neural stem cell transplantation in subacute complete spinal cord injury, with 2–4 years of follow-up including MRI, neurological and functional assessments met its primary outcome.

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Immunotherapy with a short-lived anti-PD-L1 antibody in Alzheimer’s disease: a phase 1b, randomized, double-blind trial - Nature Medicine

A phase 1b trial shows that IBC-Ab002, a short-lived antibody that intermittently activates the peripheral immune system, is safe and well tolerated with trends in biomarkers in people with early Alzheimer’s disease, supporting further clinical development.

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Immune aging biomarkers for clinical trials - Nature Medicine

The authors present a framework for the selection of immune aging biomarkers suitable for clinical trials in geroscience, alongside a translational roadmap for biomarker development to support the next generation of clinical studies on immune aging.

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A second chance for gene therapy to restore natural hearing - Nature Medicine

The immune-privileged environment of the inner ear permits sequential bilateral gene therapy for one form of congenital deafness, which adds to growing evidence of the feasibility, safety and efficacy of this innovative curative treatment.

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Lassa fever countermeasures gather pace - Nature Medicine

Two early phase clinical trials represent progress in Lassa fever vaccine and therapeutic development, including the first interventional treatment trial in decades.

dlvr.it

A neuroprosthesis for restoring hand movement and sensation in a person with complete tetraplegia - Nature Medicine

A bidirectional sensorimotor neuroprosthetic system that decodes brain signals associated with movement intention while delivering patterned neuromodulation to the spinal cord and cortex provides long-term recovery of hand motor and sensory function, even when the device is off.

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Biological aging clocks in health and disease - Nature Medicine

This review outlines the state of the art of biological clocks and how they could help understand pathobiology, reduce the burden of disease and extend healthspan.

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Immunotherapy with a short-lived anti-PD-L1 antibody in Alzheimer’s disease: a phase 1b, randomized, double-blind trial - Nature Medicine

A phase 1b trial shows that IBC-Ab002, a short-lived antibody that intermittently activates the peripheral immune system, is safe and well tolerated with trends in biomarkers in people with early Alzheimer’s disease, supporting further clinical development.

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Immune aging biomarkers for clinical trials - Nature Medicine

The authors present a framework for the selection of immune aging biomarkers suitable for clinical trials in geroscience, alongside a translational roadmap for biomarker development to support the next generation of clinical studies on immune aging.

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Shifting the goalposts in obesity drug development - Nature Medicine

Obesity drug development must move beyond an emphasis on ever-greater weight loss to prioritize tolerability, sustainable benefits and medication persistence; two recent trials exemplify this shift, with more underway.

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Human embryonic stem cell-derived dopaminergic cells for Parkinson’s disease: a phase 1/2 open-label trial - Nature Medicine

A phase 1/2 trial of an off-the-shelf stem cell-derived dopaminergic progenitor therapy demonstrated a favorable 12-month safety profile, with no graft-related adverse events, dyskinesias or tumor formation, although risks were associated with the immunosuppression regimen.

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Multi-antigen-targeting T cells in pediatric central nervous system tumors: a phase 1 trial - Nature Medicine

In the phase 1 ReMIND trial of tumor-associated antigen-specific T cells in patients with pediatric central nervous system tumors, treatment was generally well tolerated with one complete response and three long-term responders.

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Clinical decision support in hematological malignancies using a case-grounded AI agent - Nature Medicine

A locally deployable, case-grounded large language model agent achieved high concordance with hematology tumor board decisions across retrospective, external and prospective evaluations.

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Long-term health risks of e-cigarettes after smoking cessation - Nature Medicine

Real‑world evidence from a nationwide cohort suggests that using electronic cigarettes after smoking cessation may attenuate the benefits of quitting in reducing lung cancer risk and mortality; more studies are urgently needed to inform evidence-based guidance.

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Adaptive brain stimulation 2.0 for Parkinsonian gait - Nature Medicine

Two studies show that adaptive neuromodulation can improve Parkinsonian gait by matching stimulation to the right circuit at the right moment, pointing toward strategies that decompose complex symptoms into targetable spatial and temporal components.

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Immersive virtual reality with synchronous neurostimulation for upper-limb recovery after stroke: a randomized feasibility trial - Nature Medicine

A multimodal platform combining immersive virtual reality with a synchronous sensory neurostimulation protocol outperformed conventional rehabilitation in individuals with chronic stroke, driving upper-limb motor gains, restored body representation and improved tactile acuity while enabling objective kinematic tracking of recovery.

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Re-administration of AAV-mediated gene therapy for OTOF-related deafness: a single-arm trial - Nature Medicine

As part of a clinical trial, re-administration of AAV gene therapy in four patients with congenital deafness, who had previously received a first gene therapy dose and developed neutralizing antibodies, was safe and led to further hearing improvements.

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Generative AI-enabled clinical decision support system in primary care: a pragmatic, cluster-randomized trial - Nature Medicine

A pragmatic cluster-randomized trial found that ChatGPT-4o-assisted decision support in Kenyan primary care facilities did not significantly reduce 14-day treatment failure over usual care.

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Cliramitug for depletion of cardiac amyloid transthyretin: long-term follow-up of the NI006-101 trial - Nature Medicine

In an open-label extension study of the NI006-101 trial, in which patients with transthyretin amyloidosis cardiomyopathy were treated with the monoclonal antibody cliramitug, which targets misfolded transthyretin, long-term follow-up for a median of 29.3 months showed favorable safety, further reduction in cardiac amyloid burden and improvements in structural, functional and biomarker endpoints.

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CAR T cells take on precancers - Nature Medicine

CAR T cell therapy can induce deep responses but also severe toxicities in patients with smoldering myeloma, an asymptomatic cancer precursor; given the potential risks and benefits, its success depends on careful patient selection.

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Evaluating the robustness and readiness of large frontier models in health AI applications - Nature Medicine

Adversarial evaluation of leading AI models uncovers gaps between benchmark success and robustness, highlighting limitations in current health AI benchmarks and their ability to capture clinically relevant performance.

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