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        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. ...

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        Seasonal malaria chemoprevention and the spread of Plasmodium falciparum quintuple-mutant parasites resistant to sulfadoxine–pyrimethamine: a modelling study

        Seasonal malaria chemoprevention (SMC) with sulfadoxine-pyrimethamine plus amodiaquine prevents millions of clinical malaria cases in children younger than 5 years in Africa's Sahel region. However, Plasmodium falciparum parasites partially resistant to sulfadoxine-pyrimethamine (with quintuple mutations) potentially threaten the protective effectiveness of SMC. We evaluated the spread of quintuple-mutant parasites and the clinical consequences.  ...

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        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. ...

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        Interorganelle phospholipid communication, a house not so divided

        The presence of membrane-bound organelles with specific functions is one of the main hallmarks of eukaryotic cells. Organelle membranes are composed of specific lipids that govern their function and interorganelle communication. Discoveries in cell biology using imaging and omic technologies have revealed the mechanisms that drive membrane remodeling, organelle contact sites, and metabolite exchange. ...

        Research

        Workforce training needs to address social and emotional wellbeing in home-based Aboriginal and Torres Strait Islander aged care

        To explore the training needs of the home care workforce in supporting the social and emotional wellbeing of Aboriginal and Torres Strait Islander peoples receiving aged care services through the Home Care Package Program. ...

        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

        Early childhood development strategy for the world's children with disabilities

        Early childhood is foundational for optimal and inclusive lifelong learning, health and well-being. Young children with disabilities face substantial risks of sub-optimal early childhood development, requiring targeted support to ensure equitable access to lifelong learning opportunities, especially in low- and middle-income countries.  ...

        Research

        Closed Loop Study - Multicentre

        Tim Liz Jones Davis TW EA MBBS DCH FRACP MD MBBS FRACP PhD Co-head, Diabetes and Obesity Research Head, Chronic & Severe Diseases Research Focus Area; Clinical Lead, Diabetes and Obesity Research Co-head, Diabetes and Obesity Research Head, Chronic ...

        Research

        Western Australian DNA and Longitudinal Serum Bank for Weight Regulation

        Liz Tim Davis Jones EA TW MBBS FRACP PhD MBBS DCH FRACP MD Head, Chronic & Severe Diseases Research Focus Area; Clinical Lead, Diabetes and Obesity Research Co-head, Diabetes and Obesity Research Head, Chronic & Severe Diseases Research Focus Area; ...

        Research

        The impact of Influenza infection during early life on immune development

        Deborah Strickland DH PhD Program Head, Immunobiology and Immunotherapeutic Program Deb.Strickland@telethonkids.org.au Program Head, Immunobiology and Immunotherapeutic Program Associate Prof Strickland completed her PhD at the University of WA in ...

        Research

        Consensus guidelines for the diagnosis and management of invasive aspergillosis, 2021

        Invasive aspergillosis (IA) in haematology/oncology patients presents as primary infection or breakthrough infection, which can become refractory to antifungal treatment and has a high associated mortality. Other emerging patient risk groups include patients in the intensive care setting with severe respiratory viral infections, including COVID-19. ...

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