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The Phase 3 CIFFREO trial found that the gene therapy fordadistrogene movaparvovec did not significantly improve motor function in boys with Duchenne muscular dystrophy compared to placebo, leading to the discontinuation of its clinical dev
A UK multicentre study demonstrates that 24-weekly Decapeptyl SR is as effective and well-tolerated as the 12-weekly regimen for central precocious puberty, offering improved patient satisfaction and reduced clinical burden.
The ENDIA cohort study reveals that persistent single ZnT8 autoantibodies detected only via ELISA after age 4 do not significantly increase Type 1 Diabetes risk, whereas earlier detection across multiple assay formats signals higher progres
Researchers have discovered that mutations in the SNUPN gene cause spinocerebellar atrophy by disrupting RNA splicing in Purkinje cells, expanding the understanding of neurodevelopmental disorders beyond muscular conditions.
This phase 1 study evaluates the safety of placenta-derived mesenchymal stem cells (PMSCs) seeded on an extracellular matrix for in-utero repair of myelomeningocele, demonstrating successful surgical outcomes and no cell-related adverse eve
The RAAINBOW phase II/III trial demonstrates that Cinainu, a topical botanical solution, significantly improves hair regrowth and quality of life in paediatric patients with moderate-to-severe alopecia areata, offering a well-tolerated alte
This study of 100 pediatric patients with moderate-to-severe alopecia areata demonstrates that unorthodox dosing regimens of tofacitinib and baricitinib provide comparable efficacy and high tolerability over 24 weeks.
This multi-center study demonstrates that serial monitoring of plasma GFAP and NfL biomarkers can detect acute brain injury in pediatric ECMO patients before imaging diagnosis, significantly correlating with long-term survival and functiona
A prospective study demonstrates that rising levels of plasma GFAP and NfL precede neuroimaging-confirmed brain injury in children on ECMO. These biomarkers are also strongly associated with mortality and long-term functional impairment, of
This review synthesizes practice-changing pediatric research, highlighting how device selection in acute wheezing, automated oxygen titration in neonates, and novel siRNA therapies for hypercholesterolemia are redefining clinical standards
A randomized, double-blind trial in Kenya and Uganda reveals that a 500 IU fractional dose of yellow fever vaccine is not non-inferior to the standard dose in infants, suggesting that lower dose requirements in adults are not generalizable
The CD-HOPE trial reveals that cyclic exclusive enteral nutrition (C-EEN) is superior to daily partial enteral nutrition (PEN) for maintaining drug-free remission in children with Crohn’s disease, providing a critical alternative to long-te
The ORION-16 trial demonstrates that inclisiran, a twice-yearly siRNA therapy, significantly and safely reduces LDL cholesterol in adolescents with HeFH, offering a potent new tool for early cardiovascular risk management in high-risk pedia
The Phase 3 CIFFREO trial for the gene therapy fordadistrogene movaparvovec failed to meet its primary efficacy endpoint in boys with Duchenne muscular dystrophy, showing no significant functional improvement over placebo and leading to the
Explore how standardized health questionnaires empower children and parents to share their priorities in medical settings, leading to more personalized and effective treatment plans.
A Phase 1-2 trial of AAV9-GLB1 gene therapy in children with Type II GM1 gangliosidosis demonstrates significant biochemical improvements and stabilization of neurodevelopmental decline, marking a pivotal step toward treating this fatal lys
A randomized clinical trial demonstrates that Kangaroo care significantly reduces pain-related stress and systemic inflammatory responses in preterm infants undergoing retinopathy screening, suggesting a simple, non-pharmacological interven
This study highlights ultrasound viscoelastic imaging as a high-performance, noninvasive tool for diagnosing Henoch-Schönlein purpura nephritis (HSPN) in children, achieving a combined AUC of 0.95 and providing critical insights into renal
The Baby-OSCAR trial demonstrates that early selective treatment of large patent ductus arteriosus with ibuprofen in extremely preterm infants does not significantly reduce death or bronchopulmonary dysplasia, nor does it improve neurodevel
The TREOCAPA randomized clinical trial demonstrates that while prophylactic intravenous acetaminophen effectively promotes patent ductus arteriosus closure in extremely preterm infants, it does not significantly increase survival without se
