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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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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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Effects of dog ownership on children’s social-emotional development: findings from the PLAYCE cohort studyDog ownership is common in families with children and could play a role in children's social-emotional development. This study used longitudinal data on dog ownership and changing dog ownership to investigate their effects on young children's social-emotional development.
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Epigenome-Wide Association Studies of Chronic Obstructive Pulmonary Disease and Lung Function: A Systematic ReviewChronic obstructive pulmonary disease (COPD) results from gene-environment interactions over the lifetime. These interactions are captured by epigenetic changes, such as DNA methylation.
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Impact of integrating traditional care with the modern healthcare system in reducing tuberculosis diagnosis delays in Ethiopia: a clustered randomized controlled studyDiagnosis and treatment initiation delays for tuberculosis (TB) are significant challenges in resource-limited settings. These delays can result in poor treatment outcomes, disease transmission, and increased costs. This study aimed to assess the effect of integrating traditional care with modern healthcare systems on reducing TB diagnosis delay.
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Exploring Hope in Australian Justice Involved Youth with Fetal Alcohol Spectrum DisorderHope is well recognised as a positive protective factor for mental health, improved coping responses to adverse childhood events and better educational outcomes. Hope is composed of synergistic constituents – agency and pathway. A retrospective chart review was conducted of 53 justice-involved youths (10−17 years old) who underwent Fetal Alcohol Spectrum Disorder (FASD) diagnostic assessments with Patches in Western Australia between 2019 and 2020.
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Pattern of hospital admissions and costs associated with acute rheumatic fever and rheumatic heart disease in Australia, 2012–2017This study aims to describe the pattern and trends in acute rheumatic fever (ARF)/rheumatic heart disease (RHD)-related hospitalisations and costs for Australians aged <65 years.
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Sensitivity and specificity of Aboriginal-developed items to supplement the adapted PHQ-9 screening measure for depression: results from the Getting it Right studyCitation: Skinner T, Brown A, Teixeira-Pinto A, et al. Sensitivity and specificity of Aboriginal-developed items to supplement the adapted PHQ-9
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Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trialCDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Ganaxolone, a neuroactive steroid, reduces the frequency of major motor seizures in children with CDD. This analysis explored the effect of ganaxolone on non-seizure outcomes.