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An mRNA-based drug aims to replace a faulty enzyme and restore metabolic function in children with propionic acidemia — with encouraging early clinical results.
In a multicenter clinical trial, patients with early-stage Parkinson’s disease treated with lixisenatide, a drug currently used for the treatment of diabetes, showed improvement in their motor scores compared with those on placebo.
The cellular and molecular mechanisms underlying the health impacts of climate change must be better understood in order to plan interventions that mitigate harm.
As evidence emerges describing the accumulation of small plastic particles in various organs and tissues of the body, a much deeper understanding of the effects of these particles on human health is urgently needed.
The US Food and Drug Administration (FDA) has approved the first drug, resmetirom, for metabolic dysfunction-associated steatohepatitis (MASH), but much work remains for the industry, practitioners and health systems so that this approval will benefit all patients.
Researchers developed an AI model that designs novel, synthesizable antibiotic compounds — several of which showed potent in vitro activity against priority pathogens.
Diversity in clinical trials must be accompanied by justice and equity, including benefits for underrepresented participants, in order to boost population health.
Nature Medicine explores the latest translational and clinical research news, with clinical trials testing glycosylated autoantigens as immunotolerance therapies.
Growing evidence shows that dietary interventions can be effective at treating or delaying some diseases, but further trials are needed for wider adoption.
Global nephrology societies have called on the WHO and health communities to tackle the growing burden of chronic kidney disease, which has been under-recognized for too long.
Deployment of mobile vaccination teams to remote communities in Sierra Leone substantially increased COVID-19 vaccine uptake, and could potentially be bundled with other health interventions.
Patients with carotid artery plaque that contain microplastics and nanoplastics were found to be at higher risk of cardiovascular events and mortality than those in whom the particles were not detected.
In rhesus macaques, treatment with an IL-15 superagonist and broad neutralizing antibodies led to durable suppression of viremia after discontinuation of antiretroviral therapy.
In healthcare, many artificial intelligence models could be used in settings other than those for which they were approved. But such off-label use must include an empirical or mechanistic evaluation to ensure patient safety.