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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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Plasma testosterone concentration is correlated with circulating immune cell abundance in transgender young people on gender-affirming hormone treatmentSex hormones, such as oestrogen and testosterone, display significant immune modulatory properties. This is highly relevant for transgender (trans) people who undergo gender-affirming hormone (GAH) treatment. However, only a limited number of studies have evaluated the immunological impact of GAH treatments, and almost none have assessed the impact in trans young people.
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Cardiac monitoring safety assessment framework for early phase group a streptococcal vaccine trialsThis paper presents a comprehensive cardiac safety framework for early clinical development of Streptococcus pyogenes (Group A Streptococcus) vaccines, endorsed by the Strep A Vaccine Global Consortium and the Australian Strep A Vaccine Initiative. Given historical concerns about vaccine-associated acute rheumatic fever, we have established standardized echocardiography protocols integrated with clinical assessment for monitoring cardiac safety in early-phase vaccine trials.
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Children's views on outdoor advertising of unhealthy food and beverages near schoolsChildren are often exposed to unhealthy outdoor food advertisements during the school commute. This exposure can have negative public health consequences given childhood weight gain has been linked to the marketing of energy-dense and nutrient-poor foods. This study aimed to explore schoolchildren's lived experiences and attitudes towards outdoor advertising surrounding their schools.
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Determining risk features for medulloblastoma in the molecular eraNick Gottardo MBChB FRACP PhD Head of Paediatric and Adolescent Oncology and Haematology, Perth Children’s Hospital; Co-head, Brain Tumour Research
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Awake Supraglottic Airway Placement in Pediatric Patients for Airway Obstruction or Difficult Intubation: Insights From an International Airway Registry (PeDI)Small case series have described awake supraglottic airway placement in infants with significant airway obstruction and difficult intubations. We conducted this study to determine outcomes when supraglottic airways were placed in awake children enrolled in the international Pediatric Difficult Intubation Registry including success of ventilation, success of tracheal intubation, and complications.
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The Vsr-like protein FASTKD4 regulates the stability and polyadenylation of the MT-ND3 mRNAExpression of the compact mitochondrial genome is regulated by nuclear encoded, mitochondrially localized RNA-binding proteins (RBPs). RBPs regulate the lifecycles of mitochondrial RNAs from transcription to degradation by mediating RNA processing, maturation, stability and translation. The Fas-activated serine/threonine kinase (FASTK) family of RBPs has been shown to regulate and fine-tune discrete aspects of mitochondrial gene expression.
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Malaria in Nepal: A Spatiotemporal Study of the Disease Distribution and Challenges on the Path to EliminationMalaria incidence (MI) has significantly declined in Nepal, and this study aimed to investigate the spatiotemporal distribution and drivers of MI at the ward level. Data for malaria cases were obtained from the National Surveillance System from 2013 to 2021. Data for covariates, including annual mean temperature, annual mean precipitation, and distance to the nearest city, were obtained from publicly available sources. A Bayesian spatial model was used to identify factors associated with the spatial distribution of MI.
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Profiling epithelial viral receptor expression in amniotic membrane and nasal epithelial cells at birthChildren with wheeze and asthma present with airway epithelial vulnerabilities, such as impaired responses to viral infection. It is postulated that the in utero environment may contribute to the development of airway epithelial vulnerabilities.