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A prospective study reveals that ultra-rare cardiomyopathy gene variants significantly increase risk for early heart failure in infants with hypoplastic left heart syndrome, highlighting potential for genetic risk stratification and precisi
This study evaluates the long-term sinonasal health and quality of life in children after removal of button batteries or magnets from the nose, highlighting favorable outcomes with prompt intervention and need for extended monitoring in sym
This study reveals that individuals with Congenital Adrenal Hyperplasia (CAH) experience early, frequent, and complex hospitalizations with higher risk of severe outcomes, especially in infancy, compared to non-CAH patients, emphasizing the
This cohort study links polygenic scores for macronutrient intake with specific food choices in children and adolescents, highlighting genetic pathways involving hypothalamic appetite regulation but limited effects on cardiometabolic outcom
A study reveals a significant secular increase in islet autoimmunity incidence among Colorado children from 1993 to 2010, particularly in those with moderate-risk HLA genotypes, highlighting evolving risk profiles beyond known environmental
A multicountry follow-up study found no significant improvement in neurodevelopmental outcomes at two years in infants with birth asphyxia whose mothers received a single 2 g oral dose of azithromycin during labor versus placebo.
This multicentre Turkish study identifies ZnT8 autoantibodies as predominant in young siblings at risk for type 1 diabetes and highlights gestational age as a protective factor, while revealing significant anxiety changes in children and pa
A phase 2 trial shows bicistronic CD19/CD22 CAR T-cell therapy yields high remission rates with durable event-free survival in pediatric relapsed/refractory B-ALL, reducing antigen-negative relapse risks and improving outcomes.
This review synthesizes recent evidence demonstrating guanabenz’s manageable safety profile and efficacy in delaying ambulatory decline in early-onset vanishing white matter disease, highlighting translational pharmacokinetic data and
This study demonstrates that initiation of elexacaftor/tezacaftor/ivacaftor (ETI) therapy in children and adolescents with cystic fibrosis improves continuous glucose monitoring (CGM) metrics, correlating with better clinical outcomes.
Large-scale GWAS highlight novel genetic loci influencing childhood blood pressure and heart rate, with polygenic risk scores predicting adult cardiometabolic outcomes, underscoring the importance of early-life genetic determinants in cardi
The VRONI study demonstrates the feasibility and effectiveness of combined biochemical and genetic screening for familial hypercholesterolaemia (FH) in children, revealing a higher FH prevalence than previously reported and supporting natio
A Danish multicentre trial demonstrates that individualized, clinically guided antibiotic treatment lasting around 3 days is as safe as the standard 5-7 days in late-preterm and term neonates with culture-negative early-onset sepsis, signif
This review synthesizes phase 2 and 3 trial data demonstrating that the pentavalent MenABCWY vaccine is immunologically non-inferior and well tolerated compared with MenB-fHbp and MenACWY-CRM vaccines, offering a simplified, effective vacci
This study examines the relationship between children’s and parents’ screen use and language development in children aged 5 to 7 years, finding parental smartphone use may slow vocabulary growth, while device ownership correlate
This large prospective cohort study found no significant association between prenatal organophosphate pesticide exposure and autism-related traits in children, after comprehensive adjustments including maternal diet and sociodemographic fac
Late-onset sepsis (LOS) incidence among very preterm infants declined from 2010 to 2017 but has risen since, driven mainly by increasing infections from non-CoNS gram-positive bacteria and gram-negative pathogens, notably Staphylococcus aur
Routine DAT offers limited additional clinical value over transcutaneous bilirubin monitoring in low-risk ABO and RhD-incompatible neonates, supporting guideline shifts towards selective DAT use and emphasizing maternal antibody screening.
This article reviews the variability and comparative effectiveness of re-induction treatments in pediatric relapsed AML, highlighting the role of gemtuzumab ozogamicin and chemotherapy backbones in improving remission rates but not overall
This study demonstrates that classifying a Functional Status Scale score of 7 as normal underestimates long-term disability in injured children, advocating for revised scoring cutoffs and routine follow-up assessments to enhance outcome det
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