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        Research

        The development, content and response process validation of a caregiver-reported severity measure for CDKL5 deficiency disorder

        CDKL5 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.  ...

        Research

        Pneumococcal Conjugate Vaccines Are Protective Against Respiratory Syncytial Virus Hospitalizations in Infants: A Population-Based Observational Study

        Pneumococcal 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. ...

        Research

        Genotype and sleep independently predict mental health in Rett syndrome: An observational study

        Rett 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. ...

        Research

        Delivery of PEGylated liposomal doxorubicin by bispecific antibodies improves treatment in models of high-risk childhood leukemia

        High-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. ...

        Research

        Biofilms and intracellular infection in otitis media

        Otitis 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. ...

        Research

        BRAF-mediated brain tumors in adults and children: A review and the Australian and New Zealand experience

        The 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. ...

        Research

        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 trial

        SHIP-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. ...

        Research

        Impact of Missing Data on the Accuracy of Glucose Metrics from Continuous Glucose Monitoring Assessed Over a 2-Week Period

        To 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. ...

        Research

        “Connection, community and convenience”: A case study of a Facebook group for fathers navigating parenthood

        Fathers remain less likely to participate in parenting interventions which can limit their ability to receive support and build their parenting capacity. The advent of social media has engendered novel opportunities for fathers to connect with, and support, one another in the form of online peer support. Growth of these online communities exemplifies the demand from fathers to relate to other fathers who are navigating parenthood. However, the benefits of membership to these communities remain unclear. ...

        Research

        The Effect of Mineral Trioxide Aggregate Obturation Levels on the Outcome of Endodontic Retreatment: An Observational Study

        No clinical studies have examined the effect of mineral trioxide aggregate (MTA) obturation levels on the outcome of endodontic retreatment. This retrospective study examined treatment outcomes in three cohorts that compared overfilling, flush filling, and underfilling after orthograde retreatment using MTA. ...

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