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        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

        Elevated leukotriene B4 and 8-isoprostane in exhaled breath condensate from preterm-born infants

        Inflammation and oxidative stress play a key role in the development of bronchopulmonary dysplasia (BPD), possibly contributing to persistent respiratory morbidity after preterm birth. We aimed to assess if inflammatory markers were elevated in exhaled breath condensate (EBC) of infants born very prematurely (< 32 weeks gestation) at 12-16 corrected months of age, and if increased levels were associated with BPD diagnosis and respiratory morbidity. ...

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        Getting to grips with invasive group A streptococcal infection surveillance in Australia: are we experiencing an epidemic?

        Asha Rosemary Bowen Wyber AC R BA MBBS DCH FRACP PhD GAICD OAM MBChB MPH FRACGP PhD Head, Healthy Skin and ARF Prevention Senior Research Fellow Head, Healthy Skin and Arf Prevention Senior Research Fellow Areas of expertise: Skin infections, ...

        Research

        Editorial: Bench to bedside: translating pre-clinical research into clinical trials for childhood brain tumors

        Nick Raelene Gottardo Endersby NG R MBChB FRACP PhD BSc (Hons) PhD Co-Head, Brain Tumour Research Brainchild Fellow; Co-Head, Brain Tumour Research nicholas.gottardo@telethonkids.org.au raelene.endersby@telethonkids.org.au Co-Head, Brain Tumour ...

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        Increasing emergency department visits for anaphylaxis in very early childhood: A canary in the coal mine

        The incidence of anaphylaxis is increasing globally in tandem with changing environmental and lifestyle factors. There is very limited data on very early childhood presentations. We aim to assess changes in rates, characteristics and management of infant anaphylaxis in a paediatric ED over a 15-year period. ...

        Research

        Randomised controlled trial of perinatal vitamin D supplementation to prevent early-onset acute respiratory infections among Australian First Nations children: the 'D-Kids' study protocol

        Globally, acute respiratory infections (ARIs) are a leading cause of childhood morbidity and mortality. While ARI-related mortality is low in Australia, First Nations infants are hospitalised with ARIs up to nine times more often than their non-First Nations counterparts. ...

        Research

        Patterns and trends of in-hospital mortality due to non-communicable diseases and injuries in Tanzania, 2006–2015

        Globally, non-communicable diseases (NCD) kill about 40 million people annually, with about three-quarters of the deaths occurring in low- and middle-income countries. This study was carried out to determine the patterns, trends, and causes of in-hospital non-communicable disease (NCD) and injury deaths in Tanzania from 2006-2015. ...

        Research

        Difficult or impossible facemask ventilation in children with difficult tracheal intubation: a retrospective analysis of the PeDI registry

        Difficult facemask ventilation is perilous in children whose tracheas are difficult to intubate. We hypothesised that certain physical characteristics and anaesthetic factors are associated with difficult mask ventilation in paediatric patients who also had difficult tracheal intubation. ...

        Research

        Safety of BCG vaccination and revaccination in healthcare workers

        BCG vaccination and revaccination are increasingly being considered for the protection of adolescents and adults against tuberculosis and, more broadly, for the off-target protective immunological effects against other infectious and noninfectious diseases. Within an international randomized controlled trial of BCG vaccination in healthcare workers (the BRACE trial), we evaluated the incidence of local and serious adverse events, as well as the impact of previous BCG vaccination on local injection site reactions (BCG revaccination). ...

        Research

        A phase 3 study of safety and immunogenicity of V114, a 15-valent pneumococcal conjugate vaccine, followed by 23-valent pneumococcal polysaccharide vaccine, in children with HIV

        To evaluate the safety and immunogenicity of V114 [15-valent pneumococcal conjugate vaccine (PCV) containing serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9 V, 14, 18C, 19A, 19F, 22F, 23F, 33F], followed by 23-valent pneumococcal polysaccharide vaccine (PPSV23) 8 weeks later, in children with HIV. ...

        Research

        Development of an International Database for a Rare Genetic Disorder: The MECP2 Duplication Database (MDBase)

        The natural history of MECP2 duplication syndrome (MDS), a rare X-linked neurodevelopmental disorder with an estimated birth prevalence of 1/150,000 live births, is poorly understood due to a lack of clinical data collected for research. Such information is critical to the understanding of disease progression, therapeutic endpoints and outcome measures for clinical trials, as well as the development of therapies and orphan products. ...

        Research

        Understanding Engagement in Digital Mental Health and Well-being Programs for Women in the Perinatal Period: Systematic Review Without Meta-analysis

        Pregnancy and the postnatal period can be a time of increased psychological distress, which can be detrimental to both the mother and the developing child. Digital interventions are cost-effective and accessible tools to support positive mental health in women during the perinatal period. ...

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