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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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Diverse diagnostic and management approaches for acute rheumatic fever in Australia and New Zealand: findings of a prospective clinical studyThis study provides new knowledge on ARF characteristics and management and highlights international variation in diagnostic and management practice.
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Antimicrobial resistance in Staphylococcus aureus and Enterococci spp. isolates from bloodstream infections in Australian children, 2013 - 2021Rising proportions of antimicrobial resistance (AMR) have been observed in both Staphylococcus aureus and Enterococcus spp. isolates.
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The Unintended Impact of COVID-19 Associated Non-Pharmaceutical Interventions on Paediatric Hospital Admissions: An Interrupted Time Series AnalysisCOVID-19 related non-pharmaceutical interventions (NPIs) disrupted global healthcare utilisation, with notable declines in infection related paediatric hospitalisations. We aimed to identify non-infectious paediatric conditions for which the incidence of hospital admissions increased during the introduction and alleviation of NPIs in 2020.
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Invasive group B streptococcal infections in Western Australia, 2000–2018Neonatal and puerperal sepsis are major manifestations of invasive group B streptococcal (Streptococcus agalactiae; iGBS) infections. International data indicate the importance of iGBS infections among non-pregnant adults.
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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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Fathers’ Self-Compassion and Child Attachment Quality: Testing a Sequential Indirect Effect via Emotion Regulation Difficulties, Loneliness and psychological distress in two paternal samplesSelf-compassion has emerged as a promising treatment target to promote healthy parent–child relationships, though mostly in maternal samples. The mechanisms through which self-compassion may optimise the father-child relationships are not yet well-established.
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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.