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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
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.
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
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
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-inform
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
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
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
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
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
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
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
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
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.
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.
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.
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
Recent trials and meta-analyses challenge assumptions about optimal initial oxygen levels for preterm resuscitation.
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
Current evidence for behavioural and service delivery interventions in childhood epilepsy is scarce and of low quality, highlighting an urgent need for rigorous, long-term trials.
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