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Caregivers' Perceptions of Clinical Symptoms, Disease Management, and Quality of Life Impact in Cases of Cyclin-Dependent Kinase-Like 5 Deficiency Disorder: Cross-Sectional Online SurveyCyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) is an ultrarare genetic condition causing developmental epileptic encephalopathy characterized by seizures and motor and intellectual disabilities. No disease-modifying therapies are available, and treatments focus mainly on symptom management to improve quality of life.
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Management and outcomes of children hospitalised with COVID-19 including incidental and nosocomial infections in Australia 2020–2023: A national surveillance studyManagement and outcomes of children hospitalised with acute SARS-CoV-2 infection may differ throughout the pandemic or with admission type (clinical COVID-19, incidental COVID-19 or nosocomial infection).
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Circulating Soluble Factors and T-Cell Subsets as Immunological Predictors of Therapy Response in Human Cutaneous LeishmaniasisHuman cutaneous leishmaniasis, a neglected tropical disease caused by Leishmania braziliensis, presents treatment challenges due to varying therapeutic responses. Current therapies often encounter limited efficacy and treatment failure, demanding a deeper understanding of immunopathogenesis and predictive markers.
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Study of tranexamic acid on post-tonsillectomy haemorrhage (STOP) pilot trialMatt Cooper BCA Marketing, BSc Statistics and Applied Statistics, PhD Manager, Biostatistics 08 6319 1723 matt.cooper@thekids.org.au Manager,
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MetaNeb Versus Usual Care During Exacerbations of Cystic Fibrosis: An RCTDuring exacerbations, when symptom and treatment burden are increased, individuals with cystic fibrosis (CF) are likely to prefer airway clearance techniques (ACTs) that require minimal effort. Therefore, in adults with CF who were hospitalised with an exacerbation, we sought to compare the effect of the MetaNeb with usual ACTs on respiratory function and expectorated sputum.
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Beneficial health effects of ultraviolet radiation: expert review and conference reportCarcinogenic effects of ultraviolet radiation (UVR) with reference to skin cancer are the basis of widely implemented recommendations to avoid sun exposure. Whether the benefits of "restrictive sun policies" outweigh their potential harms due to diminished beneficial effects of sunlight exposure remain a matter of controversy.
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Feasibility of a 2-minute eye-tracking protocol to support the early identification of autismWe tested the potential for Gazefinder eye-tracking to support early autism identification, including feasible use with infants, and preliminary concurrent validity of trial-level gaze data against clinical assessment scores. We embedded the ~ 2-min 'Scene 1S4' protocol within a comprehensive clinical assessment for 54 consecutively-referred, clinically-indicated infants (prematurity-corrected age 9-14 months).
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Protocol for delivery of intraoperative immunotherapy to mice by surgical debulking of subcutaneous tumorsPre-clinical studies developing novel therapies to prevent cancer recurrence require appropriate surgical models. Here, we present a protocol for surgical debulking of subcutaneous tumors in mice, which allows for intraoperative application of immunotherapy-loaded biomaterials.
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Presence of onco-fetal neighborhoods in hepatocellular carcinoma is associated with relapse and response to immunotherapyOnco-fetal reprogramming of the tumor ecosystem induces fetal developmental signatures in the tumor microenvironment, leading to immunosuppressive features. Here, we employed single-cell RNA sequencing, spatial transcriptomics and bulk RNA sequencing to delineate specific cell subsets involved in hepatocellular carcinoma relapse and response to immunotherapy.
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Statistical modelling under differential privacy constraints: a case study in fine-scale geographical analysis with Australian Bureau of Statistics TableBuilder dataConsistent with the principles of differential privacy protection, the Australian Bureau of Statistics artificially perturbs all count data from the Australian Census prior to its release to researchers through the TableBuilder platform. This perturbation involves the addition of random noise to every non-zero cell count followed by the suppression of small values to zero.