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The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmapIn recent years, a small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them. This pioneering field of genetic N-of-1 therapies is evolving rapidly, giving hope for the individualized treatment of people living with very rare diseases.
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Mapping residual malaria transmission in VietnamVietnam, as one of the countries in the Greater Mekong Subregion, has committed to eliminating all malaria by 2030. Declining case numbers highlight the country's progress, but challenges including imported cases and pockets of residual transmission remain. To successfully eliminate malaria and to prevent reintroduction of malaria transmission, geostatistical modelling of vulnerability (importation rate) and receptivity (quantified by the reproduction number) of malaria is critical.
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Eating and exercise experiences of Australian trans and gender diverse folks: lived experience and stakeholder perspectivesTrans individuals face elevated health risks and socio-environmental challenges, influencing their engagement in health-protective behaviors (e.g. exercise and nutrition). Despite these challenges, there is a significant gap in understanding the specific eating and exercise experiences of Australian trans adults, including barriers to healthy behaviors and healthcare experiences. This study aims to address this gap by exploring these experiences, informing targeted interventions and healthcare practices to improve health outcomes.
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Mapping tuberculosis prevalence in Africa using a Bayesian geospatial analysisWorldwide, tuberculosis (TB) remains the leading cause of death from infectious diseases. Africa is the second most-affected region, accounting for a quarter of the global TB burden, but there is limited evidence whether there is subnational variation of TB prevalence across the continent. Therefore, this study aimed to estimate sub-national and local TB prevalence across Africa.
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Delayed airway epithelial repair is correlated with airway obstruction in young adults born very pretermNasal epithelial cells from young adults with a history of very preterm birth show delayed closure following scratch-wounding. Repair correlated with lung function, suggesting epithelial barrier integrity may play a role in preterm-associated lung disease.
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Replanting the Birthing Trees: A Call to Transform Intergenerational Trauma into Cycles of Healing and NurturingAboriginal and Torres Strait Islander ways of knowing, being and doing have fostered physical, social, and emotional wellbeing for millenia, forming a foundation of strength and resilience. However, colonisation, systemic violence and discrimination—including the forced removal of Aboriginal and Torres Strait Islander children, which continues today—have disrupted this foundation, leading to compounding cycles of intergenerational and complex trauma.
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Resting Energy Expenditure and Metabolic Features in Children With Septo-Optic DysplasiaSepto-optic dysplasia (SOD) is a major cause of congenital hypopituitarism and is known to be associated with overweight and obesity in up to 44% of children. Given the role of the hypothalamus in hormonal regulation, we sought to assess the association of resting energy expenditure (REE), appetite and physical activity with SOD.
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Safety, tolerability and pharmacokinetics of subcutaneous meropenem as an alternative to intravenous administrationSubcutaneous delivery of antibiotics is a practical alternative to IV administration. Meropenem is commonly used to treat infections caused by resistant Gram-negative organisms.
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Subnational tailoring of malaria interventions to prioritize the malaria response in GuineaIn the context of high malaria burden yet limited resources, Guinea's national malaria programme adopted an innovative subnational tailoring approach, including engagement of stakeholders, data review, and data analytics, to update their malaria operational plan for 2024-2026 and identify the most appropriate interventions for each district considering the resources available.
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Bereaved parent involvement in co-designed stillbirth research: Experiences of Project EngageWhile benefits of involving consumers in research are well established, bereaved parents face unique challenges, and descriptions of their experiences with co-designed stillbirth research are lacking. The collective experience of ‘Project Engage’ involved co-designing resources to support bereaved parents’ involvement in research.