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        FDA Patient-Focused Drug Development Guidances: Considerations for Trial Readiness in Rare Developmental and Epileptic Encephalopathies

        Developmental 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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        Vitamin D and Sunlight

        In a sunny country such as Australia, it’s important to identify how to achieve the right amount of sun exposure for good health. We need to be able to harness the benefits of vitamin D and sunlight while remaining protected from the proven dangers of too much UV radiation. ...

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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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        Descriptive contents analysis of ParticiPAte CP: a participation-focused intervention to promote physical activity participation in children with cerebral palsy

        ParticiPAte CP is a participation-focused therapy intervention that is effective to increase perceived performance of physical activity participation goals in children with cerebral palsy. We aimed to characterise the contents of ParticiPAte CP using validated behaviour change frameworks. ...

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        Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse Model

        Severity and disease progression in people with Cystic Fibrosis is typically dependent on their genotype. One potential therapeutic strategy for people with specific mutations is exon skipping with antisense oligonucleotides. CFTR exon 9 is an in-frame exon and hence the exclusion of this exon would excise only 31 amino acids but not alter the reading frame of the remaining mRNA. ...

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        Influenza epidemiology in patients admitted to sentinel Australian hospitals in 2019: the Influenza Complications Alert Network (FluCAN)

        This report summarises the epidemiology of hospitalisations with laboratory-confirmed influenza during the 2019 influenza season. The Influenza Complications Alert Network (FluCAN) is a sentinel hospital-based surveillance program that operates at sites in all jurisdictions in Australia. ...

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        An unbiased exploration of the human regulatory landscape

        Timo Lassmann T BSc (Hons) MSc PhD Feilman Fellow; Program Head, Precision Health and Team Lead, Computational Biology timo.lassmann@telethonkids.org.au Feilman Fellow; Program Head, Precision Health and Team Lead, Computational Biology Timo ...

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        Overweight/obesity and other predictors of gestational diabetes among Aboriginal and non-Aboriginal women in Western Australia

        This population-based study investigated the association of BMI and other predictors with gestational diabetes mellitus among Australian Aboriginal and non-Aboriginal mothers. We conducted a state-wide retrospective cohort study that included all singleton births in Western Australia between 2012 and 2015 using population health datasets linked by the Western Australian Data Linkage Branch. ...

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        Rift Valley fever seropositivity in humans and domestic ruminants and associated risk factors in Sengerema, Ilala, and Rufiji districts, Tanzania

        Data on Rift Valley fever virus (RVFV) prevalence in urban settings and pastoral areas of Tanzania are scarce. We performed a cross-sectional study of RVFV seroprevalence and determinants in humans and animals from Ilala, Rufiji, and Sengerema districts of Tanzania. ...

        Research

        The Development and Implementation of a Culturally Safe Survey for Measuring Knowledge, Attitudes and Values around FASD and Alcohol Use During Pregnancy in a Remote Australian Aboriginal Community Setting

        Glenn Martyn Pearson Symons G MS BA (Education) PhD Candidate B.A. (Hons) PhD. Director of First Nations Strategy and Leadership Honorary Research Associate 08 6319 1672 martyn.symons@telethonkids.org.au Director of First Nations Strategy and ...

        Research

        Cystic fibrosis clinical isolates of aspergillus fumigatus induce similar muco-inflammatory responses in primary airway epithelial cells

        Aspergillus is increasingly associated with lung inflammation and mucus plugging in early cystic fibrosis disease during which conidia burden is low and strains appear to be highly diverse. It is unknown whether clinical Aspergillus strains vary in their capacity to induce epithelial inflammation and mucus production. ...

        Research

        A world of inflammation: the need for ecological solutions that co-benefit people, place and planet

        The ecology of the early environment - including microbial diversity, nutrition, nature, social interactions and the totality of exposures in the wider "exposome" - have life-long implications for all aspects of health and resilience. In particular, the emergence of "microbiome science" provides new evidence for vital relationships between biodiversity and health at every level. ...

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