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Research

The Feasibility of Personalized Endpoints in Assessing Treatment Outcomes for Rare Diseases: A Pilot Study of Goal Attainment Scaling in SCN2A-Associated

For individuals living with rare neurodevelopmental disorders, particularly those who are at the most severe end of the spectrum, standardized outcome measures may lack the sensitivity to capture small but meaningful changes. 

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Invasive fungal disease and antifungal prophylaxis in children with acute leukaemia: a multicentre retrospective Australian cohort study

Invasive fungal disease (IFD) is a common and important complication in children with acute myeloid leukaemia (AML). We describe the epidemiology of IFD in a large multicentre cohort of children with AML.

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Optimisation of the sampling method for skin microbiome studies in healthy children: a pilot cohort study

Recent interest in the diverse ecosystem of bacteria, fungi and viruses that make up the skin microbiome has led to numerous studies investigating the skin microbiome in healthy skin and in dermatological conditions. However, skin microbiome analysis is challenging due to relatively low numbers of skin microorganisms compared to mucosal sites, such as the respiratory or gastrointestinal tracts. Microbiome results are heavily influenced by sampling methods.

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Compatibility of pentoxifylline injection with syringe and inline filters

Tobias Strunk MD, PhD, FRACP Head, Neonatal Health tobias.strunk@thekids.org.au Head, Neonatal Health Clinical Professor Tobias Strunk is a

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Parental experiences of using continuous glucose monitoring in their young children with early-stage type 1 diabetes: a qualitative interview study

To explore parents' experiences of using continuous glucose monitoring in their young children with early-stage type 1 diabetes, being followed in the Australian Environmental Determinants of Islet Autoimmunity (ENDIA) study.

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Results of an Australian trial of an automated insulin delivery (AID) system and other studies support likely benefit of AID use for many Australian adults with type 1 diabetes

Less than 20% of Australians with type 1 diabetes (T1D) meet recommended glucose targets. Technology use is associated with better glycaemia, with the most advanced being automated insulin delivery (AID) systems, which are now recommended as gold-standard T1D care. Our Australian AID trial shows a wide spectrum of adults with T1D can achieve recommended targets. Other studies, including lived experience data, are supportive. Insulin pumps are not subsidised for most Australian adults with T1D. We advocate change. 

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Net Promoter Score Model for Evaluating Paediatric Medicine Acceptability: Validation and Feasibility Study

Medicine acceptability is crucial for paediatric drug development, yet its assessment remains challenging due to the multifaceted nature of sensory attributes like taste, smell, and mouthfeel. Traditional methods of acceptability evaluation often involve complex questionnaires and lack standardisation, leading to difficulties in a comparative analysis across studies.

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Phage cocktail amikacin combination as a potential therapy for bacteremia associated with carbapenemase producing colistin resistant Klebsiella pneumoniae

The increasing occurrence of hospital-associated infections, particularly bacteremia, caused by extensively drug-resistant (XDR) carbapenemase-producing colistin-resistant Klebsiella pneumoniae highlights a critical requirement to discover new therapeutic alternatives. Bacteriophages having host-specific bacteriolytic effects are promising alternatives for combating these pathogens. 

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School physical design and its relation to bullying and student well-being

The school environment profoundly influences children's development, behaviours, and attitudes. This chapter delves into the relationship between school design and architecture, and their impact on bullying, victimisation, inclusivity, and student well-being. Research underscores the significant impact of school design on student social dynamics, advocating for collaborative efforts among stakeholders to craft effective anti-bullying policies.

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Environmental impact of inhaled medicines: A Thoracic Society of Australia and New Zealand position statement

Globally, more than 1.2 billion inhalers are purchased for asthma and chronic obstructive pulmonary disease (COPD) annually. In Australia and New Zealand, pressurized metered dose inhalers (pMDIs) are the leading delivery device prescribed and pMDI salbutamol can be purchased over the counter in Australia. These inhalers are a major contributor to healthcare related greenhouse gases.