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Liver and cardiometabolic markers and conditions in a cross-sectional study of three Australian communities living with environmental per- and polyfluoroalkyl substances contaminationPer- and polyfluoroalkyl substances (PFAS) have been associated with higher cholesterol and liver function markers in some studies, but the evidence for specific cardiometabolic conditions has been inconclusive.
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The development, content and response process validation of a caregiver-reported severity measure for CDKL5 deficiency disorderCDKL5 Deficiency Disorder (CDD) is a severe X-linked developmental and epileptic encephalopathy. Existing developmental outcome measures have floor effects and cannot capture incremental changes in symptoms. We modified the caregiver portion of a CDD clinical severity assessment (CCSA) and assessed content and response-process validity.
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Pneumococcal Conjugate Vaccines Are Protective Against Respiratory Syncytial Virus Hospitalizations in Infants: A Population-Based Observational StudyPneumococcal conjugate vaccines (PCV) reduced the risk of respiratory syncytial virus (RSV) in a randomized clinical trial. We aimed to assess the real-world effectiveness of PCV on RSV-hospitalizations among Western Australian infants.
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Genotype and sleep independently predict mental health in Rett syndrome: An observational studyRett syndrome is a genetically caused neurodevelopmental disorder associated with severe impairments and complex comorbidities. This study examined predictors of anxiety and depression in Rett syndrome, including genotype.
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BRAF-mediated brain tumors in adults and children: A review and the Australian and New Zealand experienceThe mitogen-activated protein kinase (MAPK) pathway signaling pathway is one of the most commonly mutated pathways in human cancers. In particular, BRAF alterations result in constitutive activation of the rapidly accelerating fibrosarcoma-extracellular signal-regulated kinase-MAPK significant pathway, leading to cellular proliferation, survival, and dedifferentiation.
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Delivery of PEGylated liposomal doxorubicin by bispecific antibodies improves treatment in models of high-risk childhood leukemiaHigh-risk childhood leukemia has a poor prognosis because of treatment failure and toxic side effects of therapy. Drug encapsulation into liposomal nanocarriers has shown clinical success at improving biodistribution and tolerability of chemotherapy. However, enhancements in drug efficacy have been limited because of a lack of selectivity of the liposomal formulations for the cancer cells.
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Biofilms and intracellular infection in otitis mediaOtitis media (OM), middle ear infection, represents a significant burden on children, their families, and the healthcare system. OM is the major cause of hearing loss in children and if left untreated in children who suffer chronic and recurrent forms of OM, this disease can have serious life-long sequelae.
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Impact of Missing Data on the Accuracy of Glucose Metrics from Continuous Glucose Monitoring Assessed Over a 2-Week PeriodTo explore the impact of missing data on the accuracy of continuous glucose monitoring (CGM) metrics collected for a 2-week period in a clinical trial.
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Lived experiences of the diagnostic assessment process for fetal alcohol spectrum disorder: A systematic review of qualitative evidenceEarly assessment and diagnosis of FASD are crucial in providing therapeutic interventions that aim to enhance meaningful participation and quality of life for individuals and their families, while reducing psychosocial difficulties that may arise during adolescence and adulthood. Individuals with lived experience of FASD have expertise based on their own lives and family needs. Their insights into the assessment and diagnostic process are valuable for improving service delivery and informing the provision of meaningful, person- and family-centered care. To date, reviews have focused broadly on the experiences of living with FASD.
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Automatic bronchus and artery analysis on chest computed tomography to evaluate the effect of inhaled hypertonic saline in children aged 3-6 years with cystic fibrosis in a randomized clinical trialSHIP-CT showed that 48-week treatment with inhaled 7% hypertonic saline (HS) reduced airway abnormalities on chest CT using the manual PRAGMA-CF method relative to isotonic saline (IS) in children aged 3-6 years with cystic fibrosis (CF). An algorithm was developed and validated to automatically measure bronchus and artery (BA) dimensions of BA-pairs on chest CT. Aim of the study was to assess the effect of HS on bronchial wall thickening and bronchial widening using the BA-analysis.