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Research

Measuring the Burden of Epilepsy Hospitalizations in CDKL5 Deficiency Disorder

Information on the hospital service use among individuals with CDKL5 Deficiency Disorder, an ultrarare developmental epileptic encephalopathy, is limited, evidence of which could assist with service planning. Therefore, using baseline and longitudinal data on 379 genetically verified individuals in the International CDKL5 Disorder Database, we aimed to investigate rates of seizure-related and other hospitalizations and associated length of stay in this cohort.

Research

Preclinical Assessment of Dactinomycin in KMT2A-Rearranged Infant Acute Lymphoblastic Leukemia

Infants with KMT2A-rearranged B-cell acute lymphoblastic leukemia (ALL) have high rates of relapse and poor survival compared with children. Few new therapies have been identified over the past twenty years. The aim of this study was to identify existing anti-cancer agents that have the potential to be repurposed for the treatment of infant ALL.

Research

Polyamine depletion limits progression of acute leukaemia

Cancer cells are addicted to polyamines, polycations essential for cellular function. While dual targeting of cellular polyamine biosynthesis and polyamine uptake is under clinical investigation in solid cancers, preclinical and clinical studies into its potential in haematological malignancies are lacking. Here we investigated the preclinical efficacy of polyamine depletion in acute leukaemia.

Research

Maternal concentrations of perfluoroalkyl sulfonates and alterations in white matter microstructure in the developing brains of young children

Maternal exposure to per- and polyfluoroalkyl substances (PFAS) has been linked to child neurodevelopmental difficulties. Neuroimaging research has linked these neurodevelopmental difficulties to white matter microstructure alterations, but the effects of PFAS on children's white matter microstructure remains unclear.

Research

Proposed Core Outcomes After Neonatal Sepsis: A Consensus Statement

Sepsis is one of the leading causes of neonatal mortality. There is heterogeneity in the outcomes measured and reported in studies of neonatal sepsis. To address this challenge, a core outcome set (COS) for research on neonatal sepsis was needed.

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Neurodevelopmental impairment in children with Robin sequence: A systematic review and meta-analysis

Estimate the global prevalence of neurodevelopmental impairment in children with Robin sequence (RS) at one year or more of age.

Research

Implementation Fidelity of a Smartphone Application for Population-Based General Movement Assessment: The Early Moves Study

To describe the infant and maternal characteristics of the Early Moves cohort and to assess representativeness to the general population, and to evaluate the implementation fidelity of an application-based collection of General Movement Assessment (GMA) videos at writhing and fidgety age.

Research

Yarning with a remote Aboriginal community about the next steps for achieving healthy skin

Skin health is widely recognised as being important for overall good health and well-being, yet the burden of skin infections in remote Aboriginal communities remains high. This project aimed to explore if virtual support for skin health could be a strategy to reduce community barriers to skin health engagement. 

Research

#TransTok: a digital ethnographic study using content analysis to investigate transgender and gender diverse ‘for you page’ content on TikTok that may affect mental health

Transgender and gender diverse (“trans”) people are more likely to experience adverse mental health outcomes due to the social adversities that are commonly experienced. One ameliorating factor for poor mental health outcomes can be connection to community, often facilitated in online spaces such as TikTok.

Research

Designing a paediatric teleotology pilot in the UK: Outcomes from a scoping review of the literature, with insights from the Ear Portal pilot service in Perth, Western Australia

To inform the design of a United Kingdom (UK) paediatric teleotology pilot by analysing global teleotology initiatives and drawing on insights from a successful paediatric teleotology pilot service in Perth, Western Australia (Ear Portal).