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International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USAThe clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.
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Streptococcus pyogenes pharyngitis elicits diverse antibody responses to key vaccine antigens influenced by the imprint of past infectionsKnowledge gaps regarding human immunity to Streptococcus pyogenes have impeded vaccine development. To address these gaps and evaluate vaccine candidates, we established a human challenge model of S. pyogenes pharyngitis. Here, we analyse antibody responses in serum and saliva against 19 antigens to identify characteristics distinguishing 19 participants who developed pharyngitis and 6 who did not.
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Study of Children Aged Under 2 Years Admitted With RSV at Four Australian Hospitals [2021–2022]Primary aim was to review severe acute respiratory infections (SARI) hospitalisations caused by respiratory syncytial virus (RSV) in children aged < 2 years in paediatric hospitals in Australia. Secondary aims included RSV subtyping, assessing RSV seasonality and contributing to the World Health Organisation's RSV surveillance programme.
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A roadmap for understanding sulfadoxine-pyrimethamine in malaria chemopreventionMelissa Penny PhD, PD, BSc (Hons) Professor Fiona Stanley Chair in Child Health Research melissa.penny@thekids.org.au Professor Fiona Stanley Chair
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A pilot evaluation of school-based LEGO robotics therapy for autistic studentsThere is emerging evidence that LEGO® therapy is an effective way of supporting younger autistic children develop their communication and social skills. LEGO® robotics therapy - which uses the principles of LEGO® therapy applied to LEGO® robotics - may be an age-appropriate intervention to reduce anxiety and increase social skills in autistic adolescents.
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Developmental Mismatch Across Brain Modalities in Young ChildrenBrain development during the preschool period is complex and extensive and underlies ongoing behavioral and cognitive maturation. Increasing understanding of typical brain maturation during this time is critical to early identification of atypical development and could inform treatments and interventions.
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Machine learning techniques to predict diabetic ketoacidosis and HbA1c above 7% among individuals with type 1 diabetes — A large multi-centre study in Australia and New ZealandType 1 diabetes and diabetic ketoacidosis (DKA) have a significant impact on individuals and society across a wide spectrum. Our objective was to utilize machine learning techniques to predict DKA and HbA1c>7 %.
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Cause-Specific Secular Trends and Prevention Measures of Post-Neonatally Acquired Cerebral Palsy in Victoria and Western Australia 1975–2014: A Population-Based Observational StudyTo describe the timing and causes of post-neonatally acquired cerebral palsy (PNN-CP) and map the implementation of relevant preventive strategies against cause-specific temporal trends in prevalence.
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Invasive Fungal Disease in Immunocompromised Children: Current and Emerging TherapiesIn an era of expanding indications for iatrogenic immunosuppression, invasive fungal disease (IFD) remains a significant challenge in immunocompromised children, with case fatality rates ranging from 10 to 70%. Understanding of current recommendations and recent evidence is essential to guide optimal IFD management.
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Haemophilus influenzae remains the predominant otitis media pathogen in Australian children undergoing ventilation tube insertion in the PCV13 eraUnderstanding patterns of bacterial carriage and otitis media (OM) microbiology is crucial for assessing vaccine impact and informing policy. The microbiology of OM can vary with geography, time, and interventions like pneumococcal conjugate vaccines (PCVs). We evaluated the microbiology of nasopharyngeal and middle ear effusions in children living in Western Australia, 11 years following the introduction of PCV13.