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Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trialCDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Ganaxolone, a neuroactive steroid, reduces the frequency of major motor seizures in children with CDD. This analysis explored the effect of ganaxolone on non-seizure outcomes.
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Characterising Insistence on Sameness and Circumscribed Interests: A Qualitative Study of Parent PerspectivesManifestations of insistence on sameness and circumscribed interests are complex, with individuals varying considerably, not only in the types of behaviours they express, but also in terms of a behaviour's frequency, intensity, trajectory, adaptive benefits, and impacts.
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A Pilot Group Program for Parents of Trans Young People at a Specialized Pediatric Gender Diversity ServiceParental support is strongly associated with protective factors across a range of child health outcomes for trans young people. Commonly, parents report barriers to supporting their child such as lack of understanding and difficulty in accessing information and support regarding gender diversity.
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High-flow nasal oxygen for children's airway surgery to reduce hypoxaemic events: a randomised controlled trialTubeless upper airway surgery in children is a complex procedure in which surgeons and anaesthetists share the same operating field. These procedures are often interrupted for rescue oxygen therapy.
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Patient preferences for prophylactic regimens requiring regular injections in children and adolescents: A systematic review and thematic analysisAt present, limited literature exists exploring patient preferences for prophylactic treatment of acute rheumatic fever and rheumatic heart disease. Given low treatment completion rates to this treatment in Australia, where the burden of disease predominantly affects Aboriginal and Torres Strait Islander people, an improved understanding of factors driving patient preference is required to improve outcomes.
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Angiogenesis-associated pathways play critical roles in neonatal sepsis outcomesNeonatal sepsis is a major cause of childhood mortality. Limited diagnostic tools and mechanistic insights have hampered our abilities to develop prophylactic or therapeutic interventions. Biomarkers in human neonatal sepsis have been repeatedly identified as associated with dysregulation of angiopoietin signaling and altered arachidonic acid metabolism.
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The longitudinal microbial and metabolic landscape of infant cystic fibrosis: the gut-lung axisIn cystic fibrosis, gastrointestinal dysfunction and lower airway infection occur early and are independently associated with poorer outcomes in childhood. This study aimed to define the relationship between the microbiota at each niche during the first 2 years of life, its association with growth and airway inflammation, and explanatory features in the metabolome.
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Contribution of vaccination to improved survival and health: modelling 50 years of the Expanded Programme on ImmunizationWHO, as requested by its member states, launched the Expanded Programme on Immunization (EPI) in 1974 to make life-saving vaccines available to all globally. To mark the 50-year anniversary of EPI, we sought to quantify the public health impact of vaccination globally since the programme's inception.
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The respiratory health effects of acute in vivo diesel and biodiesel exhaust in a mouse modelBiodiesel, a renewable diesel fuel that can be created from almost any natural fat or oil, is promoted as a greener and healthier alternative to commercial mineral diesel without the supporting experimental data to back these claims. The aim of this research was to assess the health effects of acute exposure to two types of biodiesel exhaust, or mineral diesel exhaust or air as a control in mice.
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Health and well-being needs of Indigenous adolescents: A protocol for a scoping review of qualitative studiesImproving the health of Indigenous adolescents is central to addressing the health inequities faced by Indigenous peoples. To achieve this, it is critical to understand what is needed from the perspectives of Indigenous adolescents themselves. There have been many qualitative studies that capture the perspectives of Indigenous young people, but synthesis of these has been limited to date.