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Edaravone for the Treatment of Motor Neurone Disease: A Critical Review of Approved and Alternative Formulations against a Proposed Quality Target Product ProfileEdaravone is one of two main drugs for treating motor neurone disease (MND). This review proposes a specific quality target product profile (QTPP) for edaravone following an appraisal of the issues accounting for the poor clinical uptake of the approved IV and oral liquid edaravone formulations. This is followed by a review of the alternative oral formulations of edaravone described in the published patent and journal literature against the QTPP.
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Effects of a live versus heat-inactivated probiotic Bifidobacterium spp in preterm infants: a randomised clinical trialHeat-inactivated probiotics (HPs) may provide an effective alternative to live probiotics by avoiding their risks (eg, probiotic sepsis) while retaining the benefits. We assessed the safety and efficacy of a HP in very preterm (VP: gestation <32 weeks) infants.
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Invasive fungal disease and antifungal prophylaxis in children with acute leukaemia: a multicentre retrospective Australian cohort studyInvasive fungal disease (IFD) is a common and important complication in children with acute myeloid leukaemia (AML). We describe the epidemiology of IFD in a large multicentre cohort of children with AML.
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Pathways of Socioeconomic Disadvantage and Peer Bullying in Children and Youth: A Scoping ReviewGrowing up in socioeconomic disadvantage increases risk of peer bullying at school. Both socioeconomic status and involvement in bullying are predictive of a range of adverse developmental outcomes. However, neither (a) the mechanisms whereby disadvantage increases bullying risk nor (b) the developmental outcomes for which bullying may mediate disadvantage are clear.
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Relationships between social determinants of health and healthy body composition among Aboriginal and Torres Strait Islander youth in the Next Generation: Youth Well-being studyPete Azzopardi PhD, FRACP, MEpi, MBBS, GDipBiostats, BMedSci Head, Adolescent Health and Wellbeing Head, Adolescent Health and Wellbeing Professor
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Early moderate prenatal alcohol exposure and maternal diet impact offspring DNA methylation across speciesAlcohol consumption in pregnancy can affect genome regulation in the developing offspring but results have been contradictory. We employed a physiologically relevant murine model of short-term moderate prenatal alcohol exposure resembling common patterns of alcohol consumption in pregnancy in humans.
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Cancer Cell Biology Research in an Indigenous Childhood Cancer ContextIn Australia, cancer medicine is increasingly guided by our expanding knowledge of cancer genomics (the study of genetic information) and biology. Personalized treatments and targets are often defined by an individual’s genetic profile—known as precision cancer medicine. The translation of genomics-guided precision therapeutics from bench to bedside is beginning to produce real clinical benefits for Australians living with cancer.
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Using Focussed Ethnography to Observe and Understand the Actions and Interactions of People With Prader-Willi Syndrome When They Exercise at a Community Gym: A ProtocolExercise for people with Prader-Willi syndrome (PWS) is important for their health and wellbeing and can provide opportunities for community participation. However, they may find it difficult to participate in some contexts, such as community gyms because social and environmental barriers in these settings may compound difficulties caused by physical impairments or intellectual disability.
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Preoperative preparation of children with upper respiratory tract infection: a focussed narrative reviewThis review summarises the current evidence for the perioperative preparation in children with upper respiratory tract infections (URTI), including COVID-19 infection. URTI, including COVID-19 infection, are common and frequent in children who present for elective surgery. Children with URTI are at increased risk of perioperative respiratory adverse events.
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An Aboriginal and Torres Strait Islander adolescent model of primary health carePete Azzopardi PhD, FRACP, MEpi, MBBS, GDipBiostats, BMedSci Head, Adolescent Health and Wellbeing Head, Adolescent Health and Wellbeing Professor