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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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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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The future of paediatric obstructive sleep apnoea assessment: Integrating artificial intelligence, biomarkers, and moreAssessing obstructive sleep apnoea in children involves various methodologies, including sleep studies, nocturnal oximetry, and clinical evaluations. Previous literature has extensively discussed these traditional methods.
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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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Examining Changes in Implementation of Priority Healthy Eating and Physical Activity Practices, and Related Barriers, Over Time in Australian Early Childhood Education and Care ServicesPromoting healthy eating and physical activity in early childhood education and care is recommended within guidelines and supported by health promotion programs; however, implementation is suboptimal. Evidence suggests implementation within the sector varies over time; however, this has not been empirically examined in relation to implementation barriers.
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Mining single-cell data for cell type-disease associationsA robust understanding of the cellular mechanisms underlying diseases sets the foundation for the effective design of drugs and other interventions. The wealth of existing single-cell atlases offers the opportunity to uncover high-resolution information on expression patterns across various cell types and
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Social Support Buffers the Association Between Alcohol Use and Depression in Australian MenAlcohol use and depression are known correlates of one another, especially in men, and are influenced by a range of demographic and psychosocial factors. This study examined the association between alcohol use and depressive symptoms in a large representative cohort of Australian men. We also tested whether this association differed by men's age, household income, social support, illicit drug use and help-seeking.
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Are assisted reproductive technology pregnancies more likely to be exposed to teratogenic medication? A whole-population studyAssisted reproductive technology (ART) pregnancies are at greater risk of birth defects than non-ART pregnancies. Teratogenic medication exposure is a potential cause of birth defects that has not been compared between ART and non-ART pregnancies.
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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