Pediatrics
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Why Universal Cholesterol Screening for Familial Hypercholesterolaemia Should Wait Until Age One
A landmark Norwegian study reveals that umbilical cord LDL-C levels overlap significantly between FH and non-FH newborns, making birth screening unreliable. In contrast, LDL-C becomes highly discriminative after age one, suggesting that uni
Idursulfase Extends Survival by a Decade in Mucopolysaccharidosis II: Final Results from the 18-Year Hunter Outcome Survey
The final report of the Hunter Outcome Survey (HOS) confirms that idursulfase treatment for Mucopolysaccharidosis II provides a 10-year survival benefit and a 57.9% reduction in mortality risk compared to untreated cohorts, alongside sustai
Breakthrough in Hunter Syndrome: Brain-Penetrant Enzyme Therapy Stabilizes Cognitive Function and Clears Lysosomal Substrates
A Phase 1-2 study of tividenofusp alfa reveals a 91% reduction in CSF heparan sulfate and stabilized adaptive behavior in children with MPS II, overcoming the blood-brain barrier via transferrin receptor-mediated transport.
Inhaled Insulin for Pediatric Type 1 Diabetes: Analyzing the INHALE-1 Trial Results and Clinical Implications
The INHALE-1 trial evaluated inhaled technosphere insulin in children. While it narrowly missed the primary HbA1c noninferiority margin, the study demonstrated improved patient satisfaction, reduced weight gain, and a favorable safety profi
Lifespan Management of ADHD: Landmark Umbrella Review Identifies Optimal Interventions and Critical Evidence Gaps
A comprehensive BMJ umbrella review synthesizes evidence for ADHD interventions across the lifespan, confirming the efficacy of stimulants and non-stimulants in the short term while revealing a critical lack of high-certainty long-term data
School-Based Digital Interventions Show Promise in Reducing Adolescent Anxiety
A cluster-randomized trial of the ‘OurFutures Mental Health’ program demonstrates significant short-term reductions in anxiety symptoms among Year 8 and 9 students, highlighting the potential for scalable, digital, trauma-informed school in
Cardiac Progenitor Cells in Hypoplastic Left Heart Syndrome: Safety Established, but Efficacy Remains Elusive in the CHILD Trial
The Phase I CHILD trial demonstrates that intramyocardial injection of neonatal cardiac progenitor cells during HLHS palliation is safe but does not improve right ventricular function at one year, despite promising signals in secondary clin
Frozen Embryo Transfer and Offspring Development: Higher Birthweight Drives Increased Bone Mineralization While BMI Remains Stable
Recent evidence indicates that children conceived via frozen embryo transfer (FET) exhibit higher bone mineral content at age 7-10 compared to fresh transfer and natural conception, a difference primarily driven by higher birthweight. Conve
Stratifying Risk in Cow’s Milk Allergy: BAT Emerges as the Superior Biomarker for Severity and Threshold
The BAT2 study reveals that the Basophil Activation Test (BAT) is the only biomarker capable of accurately predicting reaction severity to baked milk and the sensitivity threshold for fresh milk, offering a transformative tool for clinical
Nirsevimab Slashing Pediatric LRTI Hospitalizations by Over 60%: Real-World Evidence Supports Universal Immunization
A comprehensive meta-analysis of over 260,000 infants confirms that nirsevimab significantly reduces respiratory syncytial virus (RSV) and lower respiratory tract infection (LRTI) hospitalizations and emergency department visits, providing
Nirsevimab Outperforms Maternal Vaccination in Reducing Severe RSV Outcomes: Insights from Comparative Real-World Evidence
Recent real-world data from the US and France indicate that while both maternal RSV vaccination and nirsevimab are effective, nirsevimab may offer superior protection against hospitalization and intensive care admission for infants in their
Acute-Onset Axonal Neuropathy Following Infection in Children with Biallelic RCC1 Variants: A Case Series
Case series reports acute axonal neuropathy post-infection in children with biallelic RCC1 variants, often misdiagnosed as Guillain-Barré syndrome, showing variable severity and highlighting impaired nucleocytoplasmic transport as pathogeni
Precision Prognostics: Automated EEG Background Analysis and 2-Year Outcomes in Neonatal Hypoxic-Ischemic Encephalopathy
This article explores how automated EEG background analysis via the Brain State of the Newborn (BSN) score provides objective, early-life prognostication for neonates with HIE, significantly improving the prediction of 2-year neurodevelopme
Real-World Implementation of RTS,S/AS01E Malaria Vaccine Reduces Severe Malaria by 58%: Evidence from the EPI-MAL-003 Phase 4 Study
This interim analysis of the phase 4 EPI-MAL-003 study demonstrates that the RTS,S/AS01E malaria vaccine significantly reduces severe malaria, malaria-related hospitalizations, and all-cause hospitalizations in real-world clinical settings
Adolescent Body Dissatisfaction as a Causal Driver of Adult Mental Health: New Insights from a UK Twin Study
A longitudinal UK twin study suggests that adolescent body dissatisfaction may causally increase the risk of eating disorders and depression in young adulthood, with significant genetic factors underlying these associations.
Optimizing Caffeine Therapy in Preterm Infants: Does Earlier Initiation Improve Respiratory Outcomes?
This article evaluates the optimal timing and indications for caffeine initiation in preterm neonates, analyzing its impact on chronic lung disease, mortality, and neurodevelopmental outcomes based on the latest 2025 Cochrane evidence.
Probiotics Cut Clostridioides difficile Risk in Antibiotic-Exposed Patients — Small Benefit, Low Certainty
A 2025 Cochrane update finds probiotics may halve the relative risk of C. difficile-associated diarrhea in antibiotic-treated patients (ARR 1.6%, NNT 65) but evidence is low certainty and applicability varies with baseline risk.
High-frequency Oscillatory Ventilation in Near‑Term and Term Infants: Inconclusive Benefit and Possible Increased Mortality — What the 2025 Cochrane Update Tells Clinicians
A 2025 Cochrane update (3 RCTs, 368 infants) finds very low- to low-certainty evidence that HFOV neither clearly reduces treatment failure nor pulmonary air leak and may increase mortality versus conventional ventilation in term/near‑term i
Rethinking Oxygen Strategies for Preterm Infant Resuscitation: Evidence from TORPIDO, IPD-MA, and DCC Trials
Recent trials and meta-analyses challenge assumptions about optimal initial oxygen levels for preterm resuscitation.
Immediate and Early Skin-to-Skin Contact After Birth: What the 2025 Cochrane Update Means for Practice
A clear, evidence-based summary of the 2025 Cochrane review on immediate/early skin-to-skin contact (SSC), its impact on breastfeeding and newborn physiology, gaps in maternal outcomes, and practical recommendations for clinical implementat
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