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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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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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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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Barriers and facilitators to mental health care access and engagement for LGBTQA+ people with psychosis: A scoping reviewLGBTQA+ individuals are at increased risk of experiencing psychosis and face barriers in accessing appropriate and timely mental health support. This scoping review maps the existing literature to identify barriers and facilitators to access and engagement to care for LGBTQA+ people across the psychosis spectrum.
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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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Functional characterization of the MED12 p.Arg1138Trp variant in females: implications for neural development and disease mechanismSeven female individuals with multiple congenital anomalies, developmental delay and/or intellectual disability have been found to have a genetic variant of uncertain significance in the mediator complex subunit 12 gene. The functional consequence of this genetic variant in disease is undetermined, and insight into disease mechanism is required.
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A meta-analytic review and conceptual model of the antecedents and outcomes of goal adjustment in response to striving difficultiesThere is growing interest in how and why individuals adjust their goals in response to difficulties encountered during goal striving and the outcomes of such adjustments; however, research on these topics is fragmented across theoretical perspectives and life domains.
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Pediatric Bronchiectasis Action Management Plan to Improve Clinical Outcomes: A Randomized Controlled TrialManaging bronchiectasis exacerbations is a priority for patients, parents, and caregivers of children with bronchiectasis. However, evidence-based strategies among the pediatric population remain limited.