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Regeneron’s DB-OTO gene therapy shows promising results in restoring natural hearing in children with profound hearing loss caused by OTOF gene mutations, with 75% achieving significant hearing improvement after a single treatment.
The phase 3 BRAVE-AA-PEDS trial demonstrates that baricitinib, a JAK inhibitor, significantly restores scalp, eyebrow, and eyelash hair in adolescents with severe alopecia areata, achieving up to 80% hair regrowth in recent-onset cases afte
This article explores the link between prenatal and early postnatal lead exposure and adult anxiety and depression, emphasizing critical exposure windows and long-term mental health implications.
This randomized trial demonstrates that eltrombopag significantly improves sustained platelet response in children with newly diagnosed ITP, offering a potential early intervention option.
This article reviews a landmark randomized trial demonstrating that bioresorbable occluders are noninferior to metallic ones in ASD closure, with implications for improved patient outcomes and device management.
A customized protective palatal obturator significantly reduces oral tissue injury during intubation in newborns undergoing cleft lip surgery, improving safety without prolonging intubation.
This study links childhood exposure to outdoor air pollution with increased allostatic load, reflecting multi-system physiological stress, whereas road traffic noise showed no significant effect.
A randomized trial found outpatient treatment of young infants with a single low-mortality-risk sign of possible serious bacterial infection is non-inferior to inpatient care, with lower mortality rates and comparable safety.
This multicountry trial demonstrates that early switch from injectable to oral antibiotics with early discharge in young infants with moderate-risk serious bacterial infections is safe and effective, with potential to optimize healthcare re
A large multicenter study finds no association between fentanyl administration and failed reduction of pediatric ileocolic intussusception, supporting its safe use for analgesia during the painful reduction procedure.
The Basophil Activation Test offers the highest accuracy in predicting allergic reactions to baked and fresh cow’s milk during oral food challenges, reducing the need for riskier diagnostic tests in children with milk allergy.
This study developed and validated machine learning models with high accuracy to predict early pediatric sepsis and septic shock within 48 hours using multisite electronic health record data in emergency departments.
This randomized trial evaluates the safety and efficacy of everolimus combined with low-dose tacrolimus versus standard immunosuppression in children 6 months post-heart transplant, highlighting comparable rejection rates, improved kidney f
This systematic review and meta-analysis evaluates the efficacy and safety of pharmacological therapies for functional constipation in children, identifying polyethylene glycol as the preferred first-line treatment based on moderate-certain
This study reveals significant late effects in childhood neuroblastoma survivors, emphasizing the need for targeted long-term follow-up care based on treatment exposures.
EU‑CAYAS‑NET issues 44 strong, evidence‑based recommendations to standardize and improve transitions from paediatric to adult survivorship care for those diagnosed with cancer at 0–21 years, emphasizing planned transfer, risk‑based follow‑u
Recent research identifies three unique biological subtypes of chronic graft-versus-host disease (cGvHD) in children post-hematopoietic cell transplantation, offering potential pathways for tailored treatments and improved outcomes.
MRD monitoring by RT-qPCR in pediatric AML with KMT2A::MLLT10 fusion identifies patients at high risk despite morphologic remission, informing prognosis and guiding therapy.
A dynamic model predicts individualized risk of major bleeding or death with or without prophylactic platelet transfusion in preterm infants, allowing tailored clinical decisions based on evolving patient status.
Aerobic exercise significantly alleviates depressive symptoms in children and adolescents, especially in diagnosed adolescents, with 590 METs-min/week at moderate intensity identified as the optimal dose.
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