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        Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trial

        CDKL5 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.  ...

        Research

        Patient-advocate-led global coalition adapting fit-for-purpose outcomes measures to assure meaningful inclusion of DEEs in clinical trials

        Existing clinical tools that measure non-seizure outcomes lack the range and granularity needed to capture skills in developmental and epileptic encephalopathy (DEE)-affected individuals who also fall in the severe to profound range of intellectual disability. This effectively excludes those with severe impairments from clinical trials, impeding the ability of sponsors to evaluate disease-modifying therapies. ...

        Research

        Efficacy thresholds and target populations for antiviral COVID-19 treatments to save lives and costs: a modelling study

        In 2023 severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) was declared endemic, yet hospital admissions have persisted and risen within populations at high and moderate risk of developing severe disease, which include those of older age, and those with co-morbidities. Antiviral treatments, currently only available for high-risk individuals, play an important role in preventing severe disease and hospitalisation within this subpopulation. ...

        Research

        The ETO2 transcriptional cofactor maintains acute leukemia by driving a MYB/EP300-dependent stemness program

        Transcriptional cofactors of the ETO family are recurrent fusion partners in acute leukemia. We characterized the ETO2 regulome by integrating transcriptomic and chromatin binding analyses in human erythroleukemia xenografts and controlled ETO2 depletion models. We demonstrate that beyond its well-established repressive activity, ETO2 directly activates transcription of MYB, among other genes. ...

        Research

        A digital intervention to support childhood cognition after the COVID-19 pandemic: a pilot trial

        Difficulties in executive functioning (EF) can result in impulsivity, forgetfulness, and inattention. Children living in remote/regional communities are particularly at risk of impairment in these cognitive skills due to reduced educational engagement and poorer access to interventions. This vulnerability has been exacerbated by the COVID-19 pandemic and strategies are needed to mitigate long-term negative impacts on EF. ...

        Research

        Overlapping Streptococcus pyogenes and Streptococcus dysgalactiae subspecies equisimilis household transmission and mobile genetic element exchange

        Streptococcus dysgalactiae subspecies equisimilis and Streptococcus pyogenes share skin and throat niches with extensive genomic homology and horizontal gene transfer possibly underlying shared disease phenotypes. ...

        Research

        Getting creative: Using art-based techniques to identify how arts organizations enhance young people’s well-being

        Mental health concerns present significant challenges for Australian youth. Arts organizations play a key role in promoting preventative mental health strate-gies through enhancing the social and emotional well-being (SEWB) of youth. However, little is known about how the arts promote SEWB and the processes and contexts through which this occurs.  ...

        Research

        First Nations Peoples’ perceptions, knowledge and beliefs regarding stillbirth prevention and bereavement practices: A mixed methods systematic review

        First Nations Peoples endure disproportionate rates of stillbirth compared with non-First Nations Peoples. Previous interventions have aimed at reducing stillbirth in First Nations Peoples and providing better bereavement care without necessarily understanding the perceptions, knowledge and beliefs that could influence the design of the intervention and implementation.  ...

        Research

        Flexible bronchoscopy insufflated and high-flow nasal oxygen pilot trial (BUFFALO protocol pilot trial)

        Hypoxaemia occurs in approximately 30% of children during anaesthesia for flexible bronchoscopy. High-flow nasal oxygen (HFNO) can prolong safe apnoea time and be used in children with abnormal airways. During flexible bronchoscopy, there is limited evidence if HFNO confers advantages over current standard practice in avoiding hypoxaemia. The aim is to investigate feasibility of HFNO use during anaesthesia for flexible bronchoscopy to reduce frequency of rescue oxygenation and hypoxaemia.  ...

        Research

        Impacts of armed conflicts on tuberculosis burden and treatment outcomes: a systematic review

        This systematic review aimed to summarise existing literature on the impacts of armed conflicts on tuberculosis burden and treatment outcomes. ...

        Research

        How can we reach long-lasting inclusive participation for all? A vision for the future

        In 2022, an international conference was held focusing on 'participation'. We shared current evidence, identified knowledge gaps and worked together to understand what new knowledge and community and practice changes were needed. This brief communication is a summary of the conference delegates' discussions.   ...

        Research

        Cohort profile: A population-based record linkage platform to address critical epidemiological evidence gaps in respiratory syncytial virus and other respiratory infections

        The Western Australia (WA) Respiratory Infections Linked Data Platform is a population-based cohort established to investigate the epidemiology of RSV and other respiratory infections in children aged 0-10 years, incorporating microbiological testing patterns, hospital admissions, emergency department presentations, and socio-demographic data. ...

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