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Burden of illness in Rett syndrome: initial evaluation of a disorder-specific caregiver surveyRett syndrome (RTT) is a severe X-linked neurodevelopmental disorder associated with multiple neurologic impairments. Previous studies have shown challenges to the quality of life of individuals with RTT and their caregivers. However, instruments applied to quantify disease burden have not adequately captured the impact of these impairments on affected individuals and their families. Consequently, an international collaboration of stakeholders aimed at evaluating Burden of Illness in RTT was organized.
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Neighbourhood walkability and body mass index in children: Evidence from the Millennium Cohort Study in WalesOverweight and obesity in children continues to increase. Yet, the role of active transport, namely walking, in mitigating these trends remains unclear. This study examined the cross-sectional association between walkability and children's Body Mass Index (BMI) and how this varies by socio-economic and lifestyle characteristics.
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Autism likelihood in infants born to mothers with asthma is associated with blood inflammatory gene biomarkers in pregnancyMothers with asthma or atopy have a higher likelihood of having autistic children, with maternal immune activation in pregnancy implicated as a mechanism. This study aimed to determine, in a prospective cohort of mothers with asthma and their infants, whether inflammatory gene expression in pregnancy is associated with likelihood of future autism.
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Short-Term Active Safety Surveillance of the Spikevax and Nuvaxovid Priming Doses in AustraliaAustralia commenced administration of the Spikevax (Moderna mRNA-1273) COVID-19 vaccine in August 2021 and Nuvaxovid (Novavax NVX-CoV2373) in January 2022. This study describes the short-term safety profile of priming doses of the Spikevax and Nuvaxovid vaccines given between September 2021 and September 2023.
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Feasibility and acceptability of implementing an evidence-based ESCALATION system for paediatric clinical deteriorationThe ESCALATION system is a novel paediatric Early Warning System that incorporates family involvement and sepsis recognition. This study aimed to assess the feasibility and iteratively refine the ESCALATION system in a variety of hospital settings in preparation for full-service implementation.
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‘I leave most of the decisions up to her:’ Gendered parenting, un/equal decision work, and responsibility for COVID-19 vaccinationVaccination scholarship focuses on how privilege, individualized choice and ‘intensive’ and ‘natural’ parenthood – often motherhood – lead people to delay or not vaccinate their children. Recently, examining parents’ vaccination responsibilities – and the inequalities in paid employment and unpaid care work underpinning them – has become important to understand COVID-19.
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Retirement, housing mobility, downsizing and neighbourhood quality - A causal investigationThis paper provides the first causal evidence on the impact of retirement on housing choices. Our empirical strategy exploits the discontinuity in the eligibility ages for state pension as an instrument for the endogenous retirement decision and controls for time-invariant individual characteristics. The results show that retirement leads to a statistically significant and sizable increase in the probability of making a residential move or the likelihood of becoming outright homeowners.
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The critical role of the bone marrow stromal microenvironment for the development of drug screening platforms in leukemiaExtensive research over the past 50 years has resulted in significant improvements in survival for patients diagnosed with leukemia. Despite this, a subgroup of patients harboring high-risk genetic alterations still suffer from poor outcomes. There is a desperate need for new treatments to improve survival, yet consistent failure exists in the translation of in vitro drug development to clinical application.
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Pharmacokinetics of PEGasparaginase in Infants with Acute Lymphoblastic LeukemiaPEGasparaginase is known to be a critical drug for treating pediatric acute lymphoblastic leukemia (ALL), however, there is insufficient evidence to determine the optimal dose for infants who are less than one year of age at diagnosis. This international study was conducted to identify the pharmacokinetics of PEGasparaginase in infants with newly diagnosed ALL and gather insight into the clearance and dosing of this population.
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Clinical practice guidelines: Their utility, dissemination and monitoring at Colonial War Memorial Hospital: A mixed methods study from FijiClinical practice guidelines (CPGs) improve patient care by standardising medical practice. However, little is known about their applicability in low-resource settings. Since 2010, Fiji has introduced guidelines to increase the application of evidence-based practice.