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Development and initial validation of the Communication Inventory Disability – Observer Reported (CID-OR): a measure of communication in CDKL5 deficiency disorderCDKL5 Deficiency Disorder (CDD) is a rare neurodevelopmental disorder characterised by early onset seizures combined with complex healthcare needs and developmental impairment that influence functional domains including communication. Communication is a high priority domain for families but currently used measures demonstrate floor effects.
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Pharmacological targeting and characterization of Voltage-Gated Sodium Channels (VGSCs) expressed in the high-grade glioma microenvironmentHigh-grade glioma (HGG) cells reactivate neurodevelopmental programs regulated by ion channels to drive tumor progression. The activity of voltage-gated sodium channels (VGSCs) is fundamental to development, a target of blood-brain barrier (BBB)-permeable FDA-approved drugs, and aids tumor advancement in several cancers. However, the contribution of VGSC activity to HGG pathology remains unknown.
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Rare Occurrence of Congenital Neuroblastoma and Tuberous SclerosisCitation: Gardner M, Shah S, Jain N, Bynevelt M. Rare Occurrence of Congenital Neuroblastoma and Tuberous Sclerosis. Pediatr Neurol. 2026;176:62-3.
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Characterising commensal and pathogenic staphylococcal interactions with neonatal and adult bloodThe abundant skin commensal, Staphylococcus epidermidis, is the leading cause of late-onset sepsis (LOS) in preterm infants but rarely causes infections in term infants and adults. Staphylococcal virulence mechanisms and the role of the preterm immune responses in driving these life-threatening infections remain poorly understood.
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Children's Anxiety in the Perioperative Environment: A Qualitative Exploration With Children, Parents and Staff at a Tertiary Paediatric HospitalPerioperative anxiety is a common and distressing aspect of anaesthesia for many children, resulting in management challenges at the time of anaesthesia and potential physical and psychological adverse outcomes. We conducted this qualitative phenomenological study to explore the perspectives of children, parents and staff on perioperative anxiety in our institution. Planned recruitment was 20 each of children who had undergone elective anaesthesia, their parents and staff.
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Mapping Bacillus Calmette-Guérin vaccination coverage in Africa from 1990 to 2022: a novel spatiotemporal modelling studyBacillus Calmette-Guérin (BCG) protects children from severe tuberculosis and remains the only licensed vaccine for tuberculosis. Subnational estimates of BCG coverage are essential for identifying underserved populations across Africa. This study aimed to map BCG vaccination coverage in Africa from 1990 to 2022.
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Parents’ Work–Family Conflict and Children’s Emotional Well-Being: The Mediating Role of Parenting BehaviorsDespite growing evidence that parental work–family conflict (WFC) affects children’s emotional well-being, little is known about the multiple pathways underlying such effects. This study examines the association between parental WFC and children’s emotional well-being and the potential mediating role of parenting behaviors in this process.
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Microbiota-derived butyrate promotes a FOXO1-induced stemness program and preserves CD8+ T cell immunity against melanomaA range of microbiota species correlate with improved cancer outcomes in patients and confer protection in pre-clinical mouse models. Here, we examined how microbiota regulate CD8+ T cell immunity against melanoma. Spontaneous control of cutaneous melanoma in mice correlated with metabolic pathways required for microbial synthesis of short-chain fatty acids (SCFAs) shared between several microbiota species.
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Value profile for Malaria vaccines and monoclonal antibodies1Malaria remains a leading cause of morbidity and mortality and is responsible for over 0.5 million annual deaths globally. During the first two decades of this century, scale-up of a range of tools was associated with significant reductions in malaria mortality in the primary risk group, young African children.
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Beyond Seizures as an Outcome Measure: A Global Severity Scoring System for CDKL5 Deficiency DisorderCDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy (DEE) associated with multiple impairments and comorbidities. Outcome measures for disease-modifying clinical trials for DEEs should measurably capture a spectrum of caregiver priorities and be externally validated.