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Research

Rett 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.

Research

Children receiving treatment for acute myeloid leukemia (AML) are at high risk of invasive fungal disease (IFD). Evidence from pediatric studies support the efficacy of antifungal prophylaxis in reducing the burden of IFD in children receiving therapy for AML, yet existing antifungal agents have specific limitations and comparative data to inform the optimal prophylactic approach are lacking.

Research

Mothers 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. 

Research

Australia 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. 

Research

The 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.

Research

Vaccination 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.

Research

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

Globally, Indigenous peoples have incurred significant harm due to colonisation of their lands. Dispossession of culture, language, family and land, and the historical, systematic removal of children in Australia (the ‘Stolen Generation’), has resulted in evident ongoing negative outcomes in the contemporary lives of Aboriginal and Torres Strait Islander people.

Research

Platinum-based chemotherapy in combination with anti-PD-L1 antibodies has shown promising results in mesothelioma. However, the immunological mechanisms underlying its efficacy are not well understood and there are no predictive biomarkers to guide treatment decisions.

Research

Drug-resistant tuberculosis (DR-TB) remains a major public health challenge in China, with varying treatment outcomes across different regions. Understanding the spatial distribution of DR-TB treatment outcomes is crucial for targeted interventions to improve treatment success in high-burden areas such as Hunan Province. This study aimed to map the spatial distribution of DR-TB treatment outcomes at a local level and identify sociodemographic and environmental factors associated with poor treatment outcomes in Hunan Province, China.