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Edaravone for the Treatment of Motor Neurone Disease: A Critical Review of Approved and Alternative Formulations against a Proposed Quality Target Product ProfileEdaravone is one of two main drugs for treating motor neurone disease (MND). This review proposes a specific quality target product profile (QTPP) for edaravone following an appraisal of the issues accounting for the poor clinical uptake of the approved IV and oral liquid edaravone formulations. This is followed by a review of the alternative oral formulations of edaravone described in the published patent and journal literature against the QTPP.
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Effects of a live versus heat-inactivated probiotic Bifidobacterium spp in preterm infants: a randomised clinical trialHeat-inactivated probiotics (HPs) may provide an effective alternative to live probiotics by avoiding their risks (eg, probiotic sepsis) while retaining the benefits. We assessed the safety and efficacy of a HP in very preterm (VP: gestation <32 weeks) infants.
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Septo-optic dysplasia and gastroschisis: trends in birth prevalence and association with maternal ageThis study aims to describe the risk factors and trends in birth prevalence of septo-optic dysplasia (SOD) and gastroschisis between 1980 and 2023. This descriptive, population-based study of SOD and gastroschisis used Western Australian Register of Developmental Anomalies data from 1980 to 2023. Birth prevalence was calculated using Midwives Notification System data for all births after 20 weeks gestation.
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Parental experiences of using continuous glucose monitoring in their young children with early-stage type 1 diabetes: a qualitative interview studyTo 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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Net Promoter Score Model for Evaluating Paediatric Medicine Acceptability: Validation and Feasibility StudyMedicine 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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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 diabetesLess 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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Phage cocktail amikacin combination as a potential therapy for bacteremia associated with carbapenemase producing colistin resistant Klebsiella pneumoniaeThe 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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Compatibility of pentoxifylline injection with syringe and inline filtersTobias 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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School physical design and its relation to bullying and student well-beingThe 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 statementGlobally, 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.