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FDA Patient-Focused Drug Development Guidances: Considerations for Trial Readiness in Rare Developmental and Epileptic EncephalopathiesDevelopmental and epileptic encephalopathies (DEE) are rare, often monogenic neurodevelopmental conditions. Most affected individuals have refractory seizures. All have multiple severe impairments which can be as life-limiting as or more limiting than the seizures themselves. Mechanism- and gene-targeted therapies for these individually rare, genetic conditions hold hope for treatment, amelioration of disease expression, and even cure.
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Neuropsychological profiles of adolescents sentenced to detention in Western Australia with and without prenatal alcohol exposureYouth with prenatal alcohol exposure (PAE) are under-recognised in the justice system, warranting improved identification. This study aimed to compare neuropsychological profiles of adolescents, with and without PAE and identify neuropsychological tasks predictive of PAE-group membership. It was hypothesised that participants with PAE would score significantly lower on neuropsychological tests.
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Airway management in the paediatric difficult intubation registry: a propensity score matched analysis of outcomes over timeThe Paediatric Difficult Intubation Collaborative identified multiple attempts and persistence with direct laryngoscopy as risk factors for complications in children with difficult tracheal intubations and subsequently engaged in initiatives to reduce repeated attempts and persistence with direct laryngoscopy in children. We hypothesised these efforts would lead to fewer attempts, fewer direct laryngoscopy attempts and decrease complications.
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An Analysis of the Gut Microbiota and Related Metabolites following PCSK9 Inhibition in Statin-Treated Patients with Elevated Levels of Lipoprotein(a)Atherosclerotic cardiovascular disease (ASCVD) is a leading cause of global mortality, often associated with high blood levels of LDL cholesterol (LDL-c). Medications like statins and PCSK9 inhibitors, are used to manage LDL-c levels and reduce ASCVD risk. Recent findings connect the gut microbiota and its metabolites to ASCVD development.
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Hybrid Closed-Loop Versus Manual Insulin Delivery in Adults With Type 1 Diabetes: A Post Hoc Analysis Using the Glycemia Risk IndexGlycemia risk index (GRI) is a novel composite metric assessing overall glycemic risk, accounting for both hypoglycemia and hyperglycemia and weighted toward extremes. Data assessing GRI as an outcome measure in closed-loop studies and its relation with conventional key continuous glucose monitoring (CGM) metrics are limited.
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Mosaic Darier disease flaring following allogeneic stem cell transplantBernadette Ricciardo MBBS (hon) DCH FACD PhD Candidate Bernadette.Ricciardo@thekids.org.au PhD Candidate Dr Bernadette Ricciardo is a PhD student on
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Outcomes of latent rheumatic heart disease: External validation of a simplified score in patients with and without secondary prophylaxisSecondary antibiotic prophylaxis reduces progression of latent rheumatic heart disease (RHD) but not all children benefit. Improved risk stratification could refine recommendations following positive screening. We aimed to evaluate the performance of a previously developed echocardiographic risk score to predict mid-term outcomes among children with latent RHD.
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BreastfeedingConvenient, readily available and helping create a close and loving bond between baby and mother, breastfeeding is highly regarded for optimising infant health and preventing chronic disease in adulthood.
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Performance characteristics and potential public health impact of improved pre-erythrocytic malaria vaccines targeting childhood burdenNew malaria vaccine development builds on groundbreaking recommendations and roll-out of two approved pre-erythrocytic vaccines (PEVs); RTS,S/AS01 and R21/Matrix-M. Whilst these vaccines are effective in reducing childhood malaria within yearly routine immunization programs or seasonal vaccination, there is little evidence on how different PEV efficacies, durations of protection, and spacing between doses influence the potential to avert uncomplicated and severe childhood malaria.
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Profile of the PLAY spaces & environments for children’s physical activity, sedentary behaviour and sleep (PLAYCE) cohort study, Western AustraliaChildhood is a critical period for the development of movement behaviours such as physical activity, sleep and sedentary behaviour. The PLAYCE Cohort was established to investigate how movement behaviours change over early to middle childhood, across key behaviour settings and relationships with health and development. An overview of the PLAYCE cohort, summary of key findings to date, and future research opportunities are presented.