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A novel, palatable paediatric oral formulation of midazolam: Pharmacokinetics, tolerability, efficacy and safetyWe conclude that the novel chocolate-based formulation of midazolam provides improved tolerability while remaining efficacious
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Research priority setting in childhood chronic disease: a systematic reviewResearch priority setting activities in paediatric chronic disease cover many discipline areas and have elicited a broad range of topics
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Predicting respiratory hospital admissions in young people with cerebral palsyMost risk factors for respiratory hospital admissions in young people with cerebral palsy are potentially modifiable
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A group randomized controlled trial evaluating parent involvement in whole-school actions to reduce bullyingWhole-school capacity-building intervention in early and middle childhood can improve the likelihood and frequency of positive parent–child communication about bullying
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SToP (See, Treat, Prevent) skin sores and scabies trial: study protocol for a cluster randomised, stepped-wedge trial for skin disease control in remote Western AustraliaSkin infection burden in remote Aboriginal communities can be reduced by the See, Treat, Prevent (SToP skin sores and scabies) trial
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Psychometric properties of the Quality of Life Inventory-Disability (QI-Disability) measureInitial evaluation suggests that QI-Disability is a reliable and valid measure of quality of life across the spectrum of intellectual disability
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International experience in the development of patient-derived xenograft models of diffuse intrinsic pontine gliomaThis multi-center study provides valuable information on the success rate of establishing patient-derived pre-clinical models of diffuse intrinsic pontine glioma
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Maternal high fat diet compromises survival and modulates lung development of offspring, and impairs lung function of damsWhile maternal high fat diet compromised litter survival, it also promoted somatic and lung growth (increased lung volume) in the offspring
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C1 CAGE detects transcription start sites and enhancer activity at single-cell resolution.We introduce C1 CAGE, a method for the detection of transcript 5'-ends with an original sample multiplexing strategy in the C1TM microfluidic system
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A Diagnosis for All Rare Genetic Diseases: The Horizon and the Next FrontiersThere is a critical need for the field to develop and implement strategies to understand the mechanisms underlying all rare diseases and translate these to clinical care