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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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Hearing loss in Australian First Nations children at 6-monthly assessments from age 12 to 36 months: Secondary outcomes from randomised controlled trials of novel pneumococcal conjugate vaccine schedulesIn Australian remote communities, First Nations children with otitis media (OM)-related hearing loss are disproportionately at risk of developmental delay and poor school performance, compared to those with normal hearing. Our objective was to compare OM-related hearing loss in children randomised to one of 2 pneumococcal conjugate vaccine (PCV) formulations.
Tourette syndrome is a neurodevelopmental disorder characterised by uncontrollable movements and vocalisations known as tics.
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Increasing airway obstruction through life following bronchopulmonary dysplasia: a meta-analysisFew studies exist investigating lung function trajectories of those born preterm; however growing evidence suggests some individuals experience increasing airway obstruction throughout life. Here we use the studies identified in a recent systematic review to provide the first meta-analysis investigating the impact of preterm birth on airway obstruction measured by the forced expiratory volume in 1 s (FEV1) to forced vital capacity (FVC) ratio.
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Reduced socs1 expression in lung fibroblasts from patients with ipf is not mediated by promoter methylation or mir155The interleukin (IL)-6 family of cytokines and exaggerated signal transducer and activator of transcription (STAT)3 signaling is implicated in idiopathic pulmonary fibrosis (IPF) pathogenesis, but the mechanisms regulating STAT3 expression and function are unknown. Suppressor of cytokine signaling (SOCS)1 and SOCS3 block STAT3, and low SOCS1 levels have been reported in IPF fibroblasts and shown to facilitate collagen production. Fibroblasts and lung tissue from IPF patients and controls were used to examine the mechanisms underlying SOCS1 down-regulation in IPF.
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Feasibility of upper airway collapsibility measurements in anesthetized childrenPatients with a propensity for upper airway obstruction, including those with obstructive sleep apnea (OSA), are vulnerable in the perioperative period. OSA is an increasingly common disorder in children and, when present, is associated with an increased risk of perioperative respiratory adverse events (PRAE),1 morbidity, and mortality. Therefore, identifying at-risk patients is vital to provide tailored perioperative anesthetic management.
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Reporting Both Unadjusted and Adjusted Estimates Is Essential to the Interpretation of Randomized Clinical Trial Results - ReplyAndrew Matt Videos Whitehouse Watch and listen to Andrew Cooper PhD BCA Marketing, BSc Statistics and Applied Statistics, PhD Deputy Director (
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A Rationale for Change: An Increase in Invasive Pneumococcal Disease in Fully Vaccinated ChildrenA review of cases informed a change from a "3 + 0" infant schedule to a "2 + 1" schedule
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Birth outcomes in Aboriginal mother–infant pairs from the Northern Territory, Australia, who received 23-valent polysaccharide pneumococcal vaccination during pregnancyWe found a numerically higher rate of preterm births among women who received 23vPPV in pregnancy compared to unvaccinated pregnant women
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Evaluation of the Learning+ Pilot ProgramYasmin Adrienne Harman-Smith Gregory BA, BHlthSc(Hons), PhD BSocSci, MPhil Head, Early Years Systems Evidence; Head, Tenders Support Unit Project