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Predictive gene expression signature diagnoses neonatal sepsis before clinical presentationNeonatal sepsis is a deadly disease with non-specific clinical signs, delaying diagnosis and treatment. There remains a need for early biomarkers to facilitate timely intervention. Our objective was to identify neonatal sepsis gene expression biomarkers that could predict sepsis at birth, prior to clinical presentation.
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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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The Respiratory Microbiome in Paediatric Chronic Wet Cough: What Is Known and Future DirectionsChronic wet cough for longer than 4 weeks is a hallmark of chronic suppurative lung diseases, including protracted bacterial bronchitis, and bronchiectasis in children. Severe lower respiratory infection early in life is a major risk factor of PBB and paediatric bronchiectasis.
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Young adult outcomes following premature birth: A Western Australian experienceChildhood outcomes following preterm birth are widely published, however long-term adult outcomes are less well described. We aimed to determine the quality of life and burden of co-morbidities experienced by preterm-born young adults in Western Australia.
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The impact for DCD – USA study: The current state of Developmental Coordination Disorder (DCD) in the United States of AmericaDevelopmental Coordination Disorder (DCD) is among the most under-recognized and under-supported disorders worldwide. The aim of this study was to present a preliminary national study that evaluated the unmet needs of children with DCD in the USA using the Impact for DCD survey.
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Investigating the impact of autistic children's feeding difficulties on caregiversThe aim of this study was to investigate the influence of children's autism characteristics, sensory profiles and feeding difficulties on caregiver-reported impact at mealtimes.
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Caregiver-perceived racial discrimination is associated with diverse mental health outcomes in Aboriginal and Torres Strait Islander children aged 7-12 yearsExposure to racial discrimination in Aboriginal children increased the risk for a spectrum of interrelated factors linked to negative mental health
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Single Parasternal-Long-Axis-View-Sweep Screening Echocardiographic Protocol to Detect Rheumatic Heart Disease: A Prospective Study of Diagnostic AccuracyEchocardiographic screening in school-aged children can detect rheumatic heart disease (RHD) prior to the manifestation of symptoms of heart failure.
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The high prevalence and impact of rheumatic heart disease in pregnancy in First Nations populations in a high-income setting: a prospective cohort studyRheumatic heart disease in pregnancy persists in First Nations people in Australia and New Zealand and is associated with major cardiac and perinatal morbidity
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An introduction to clinical trial designThis manuscript will give a brief overview of clinical trial design including the strengths and limitations of various approaches