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An interim report from the GAIN/iCat2 study shows that molecular profiling of pediatric solid malignancies clarifies diagnostic classifications and provides opportunities for matched targeted therapy.
In a phase 2a clinical trial in patients with nonalcoholic steatohepatitis, dual inhibition of sodium–glucose cotransporters 1 and 2 with 150 mg of licogliflozin led to reductions in serum alanine aminotranferase levels.
Analysis of mutations that arise in chronic SARS-CoV-2 infections shows both overlap and differences with mutations present in pandemic viral variants of concern, highlighting their distinct drivers of evolution.
The authors propose a new conceptual model of critical illness that moves away from the current syndrome-based framework in favor of more precise biological descriptors—spurred by mounting translational evidence and insights from Coronavirus Disease 2019 (COVID-19) research.
For infants with three copies of SMN1 at risk for spinal muscular atrophy (SMA) type 1, onasemnogene abeparvovec improves ventilator-free survival and nutritional/respiratory independence and allows motor development indistinguishable from healthy children without SMA.
Restoration of visual function in degenerative retinal disease may be an attainable goal, thanks to advances in cell-based therapy, gene therapy, and prosthetics. This Review discusses the most promising strategies that will probably reach clinical use in the next decade.
For presymptomatic infants at risk for SMA type 1, onasemnogene abeparvovec improves motor outcomes, ventilator-free survival, and nutritional/respiratory independence compared with untreated or treated symptomatic patients
New technologies allow the noninvasive detection and staging of asymptomatic alcohol-associated liver disease; further refinement of this approach could transform clinical management and improve patient outcomes.
Online access during the pandemic widened participation in scientific conferences for women, young scientists and those from low- and middle-income countries, and should be continued
Patients with HER2-low metastatic breast cancer are a historically overlooked group, but a phase 3 trial reveals trastuzumab deruxtecan as a new treatment option that prolongs survival.
A new study generated and optimized a polygenic score for chronic kidney disease with reproducible performance across 15 cohorts of different ancestries, and identified potentially clinically relevant thresholds with predicted effects comparable to having a family history of the disease.
A model that predicts the effectiveness of COVID-19 vaccines against circulating SARS-CoV-2 variants, before the acquisition of real-world effectiveness data, may help guide more rapid public health and research responses to new variants of concern.
Substance addiction is a major public health crisis. Neuromodulation treatments show promise, but the therapeutic targets remain unclear. Studying human brain lesions that led to addiction remission with the lesion-network-mapping approach resulted in the identification of a brain circuit shared across addiction disorders that may have therapeutic potential.
Leading researchers explain how the COVID-19 pandemic has spurred research on wearables, genomic surveillance, behavioral interventions and drug discovery.
