Pediatrics
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Genomic Newborn Screening Is Feasible, Highly Acceptable and Clinically Actionable: Lessons from the BabyScreen+ Cohort
BabyScreen+ shows clinically accredited whole‑genome sequencing from dried blood spots can be integrated into newborn screening at scale: 1.6% of 1,000 babies had high‑chance actionable findings, median return 13 days, strong parental accep
Atosiban Did Not Improve Neonatal Outcomes for Threatened Preterm Birth at 30–34 Weeks: Results from the Multicentre APOSTEL 8 Trial
In the APOSTEL 8 randomized trial, intravenous atosiban did not reduce a composite of perinatal mortality and severe neonatal morbidity versus placebo for threatened preterm birth at 30+0–33+6 weeks.
Nirsevimab Halves RSV Bronchiolitis Risk in Infants — High Effectiveness Sustained Despite Emergent RSV‑B Mutations
A multicenter test-negative case-control study in France found high and sustained effectiveness of nirsevimab against RSV bronchiolitis in infants across two consecutive seasons (83.2% and 89.3%), despite emergence of RSV-B mutations.
Nirsevimab Cuts RSV Lower Respiratory Infections in Infants Without Replacing Other Viruses: Post‑hoc Analysis of the Phase 3 MELODY Trial
Post hoc analysis of the MELODY Phase 3 trial shows nirsevimab reduced RSV-associated lower respiratory tract infections and RSV coinfections in healthy term and late-preterm infants, with no signal of viral replacement by other respiratory
Nirsevimab in Chile’s First National RSV Campaign: 76% Reduction in RSV Hospitalisations and 85% Fewer ICU Admissions in Infants
A nationwide retrospective study from Chile finds that a universal nirsevimab immunisation strategy in 2024 reduced RSV-related lower respiratory tract infection hospitalisations by 76% and ICU admissions by 85%, with an NNT of 35 to preven
Real-world data suggest nirsevimab reduces RSV test-positivity up to 12 months but not beyond — cautious interpretation required
A multicentre retrospective TriNetX analysis found reduced odds of RSV test-positivity for infants receiving nirsevimab when last dose was within 6–11 months before testing; protection was strongest within 6 months and not evident beyond 12
Widespread Nirsevimab and Maternal RSV Vaccination Linked to Large Drop in Infant Hospitalizations in First Full Season
Real-world surveillance from 2024–2025 shows substantial reductions in RSV-associated hospitalizations among infants after broad availability of maternal RSV vaccination and nirsevimab, with effectiveness estimates approximating those from
Screening Children and Young Adults for Familial Hypercholesterolemia: Clinically Helpful but Not Cost‑Effective
A modeling study found that sequential population screening for familial hypercholesterolemia (FH) at ages 10 or 18 is clinically effective but not cost‑effective versus usual care, unless follow-up monitoring and lifestyle interventions fo
Less Sitting, More Sleep and MVPA Linked to Better Glycaemic Stability in Children with Type 1 Diabetes: A 2‑Year Compositional Analysis
A longitudinal compositional analysis of 83 children with type 1 diabetes found that reallocating daily time away from sedentary behaviour toward sleep or moderate‑to‑vigorous physical activity (MVPA) was associated with lower HbA1c and int
Balance, Not Bedrest: Daily Movement Patterns Linked to Faster Symptom Recovery After Pediatric Concussion
Compositional analysis of the PedCARE cohort suggests that an early period of relative rest combined with above-average light and moderate-to-vigorous activity over the first two weeks is associated with lower postconcussion symptom burden
FDA Approves Linzess for Pediatric IBS with Constipation: A New Hope for Children
The FDA has approved linaclotide (Linzess) for treating irritable bowel syndrome with constipation (IBS-C) in children 7 and older, marking the first official treatment option for pediatric IBS-C.
Darker Skin Tone Reduces Accuracy of Cerebral NIRS in Children: Implications from a Prospective Cohort
A prospective pediatric study shows forehead NIRS (INVOS 5100C) underestimates regional oxygenation in children with darker skin, producing clinically meaningful bias and raising concerns about equity in monitoring and device validation.
Understanding the FDA’s Rejection of SYD-101: Navigating the Challenges in Pediatric Myopia Treatment
The FDA has rejected Sydnexis’s low-dose atropine eye drop SYD-101 for controlling childhood myopia progression despite promising trial results, highlighting challenges in demonstrating long-term efficacy and clinical significance in new pe
AAV‑OTOF Gene Therapy Restores Substantial Hearing in DFNB9 Across Ages — Rapid, Age‑dependent Benefits in a First-in‑Human Trial
A single‑arm trial of AAV‑OTOF (Anc80L65) in 10 patients (1.5–23.9 years) with autosomal recessive deafness 9 showed good tolerability and rapid, clinically meaningful hearing gains, with optimal outcomes in children aged 5–8 years; longer
OTOF Gene Therapy Rivaled—and in Some Domains Outperformed—Cochlear Implants in Restoring Hearing and Speech in Congenital Deafness
A 2022–2024 nonblinded cohort study found OTOF gene therapy produced rapid, durable gains in auditory thresholds, speech perception, mismatch-negativity responses, and music perception versus cochlear implantation in children with genetical
Congenital CMV in Australia: Reported Cases Are a Small Fraction of Estimated Burden — Implications for Screening, Treatment, and Public Health
National APSU surveillance (1999–2024) found 479 definite congenital CMV cases and dramatic under-ascertainment versus expected prevalence; symptomatic infants increasingly receive antivirals. Expanded surveillance, newborn screening, and c
Unilateral Pediatric Chronic Otitis Media with Effusion Impairs Binaural Hearing and Raises Tinnitus Risk: Evidence Challenging Conservative Management
A prospective cross-sectional study finds unilateral chronic otitis media with effusion (COME) in children is associated with poorer spatial release from masking and high tinnitus prevalence, suggesting potential central auditory consequenc
AI-RAPNO: Translating Artificial Intelligence into Reliable Response Assessment for Pediatric Neuro-Oncology
AI-RAPNO synthesizes current AI developments for pediatric brain tumour imaging and provides practical recommendations to improve trustworthiness, generalisability, and clinical implementation of AI-driven response assessment using the RAPN
Cochlear Implant Benefits in Children with Single‑Sided Deafness: Better in Quiet, Challenged in Noise — and Time Matters
In 185 children receiving unilateral cochlear implants, residual acoustic hearing favored the non-implanted ear in quiet (especially for SSD), but noise attenuated that advantage. Shorter auditory deprivation and longer CI experience reduce
Longitudinal Developmental Outcomes and Early Cochlear Implantation Benefits in Children with Prelingual Single-Sided Deafness: Evidence Synthesis and Clinical Recommendations
Early cochlear implantation in children with prelingual single-sided deafness improves spatial hearing and cognitive outcomes, supporting neurodevelopment and mitigating risks of auditory deprivation and cortical reorganization.
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