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Final results of two studies — whose preliminary data led to regulatory approval of the gene therapy exagamglogene autotemcel — describe highly effective treatment of sickle-cell disease and transfusion-dependent β-thalassemia.
The lay summary of trial results to be provided to participants should be written in plain language, use infographics and be concise — something that currently is almost never achieved.
The World Health Organization framework for tracking SARS-CoV-2 variants has been updated to reflect the continued evolution of the virus; this framework could be adapted for other emerging respiratory diseases with epidemic and pandemic potential.
Growing interest in non-invasive brain–computer interfaces, rather than implants, might improve accessibility for patients, but resolution needs to be improved.
A randomized controlled trial involving a telemedicine-based approach for the management of patients with acute coronary syndrome had several clinical benefits relative to standard of care.
An argument framework, grounded in the sciences of reasoning, provides an alternative to medicine’s measurement framework for evaluating and synthesizing evidence in healthcare.
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.