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The United Nations convention on rare diseases—A framework for research prioritization

Citation: Junaid M, Downs J, Groza T, Lassmann T, Baker S, et al. The United Nations convention on rare diseases—A framework for research

Convalescent plasma in hospitalised patients with COVID-19

Convalscent plasma (CP) was identified as a potential therapy for COVID-19 available early in the pandemic.

A meta-analytic review and conceptual model of the antecedents and outcomes of goal adjustment in response to striving difficulties

There is growing interest in how and why individuals adjust their goals in response to difficulties encountered during goal striving and the outcomes of such adjustments; however, research on these topics is fragmented across theoretical perspectives and life domains. 

Invasive mould infection in children – advances made or obstacles remaining?

Invasive mould infection (IMI) is a major cause of morbidity and mortality in immunocompromised children. Outcomes for paediatric patients with IMI remain poor, due in part to the limitations of available diagnostic tools and therapeutic agents.

Rethinking a hybrid malaria chemoprevention delivery strategy for children in sub-perennial settings: a modelling study integrating age- and seasonally-targeted delivery

The World Health Organization recommends perennial malaria chemoprevention (PMC), generally using sulfadoxine-pyrimethamine (SP) to children at high risk of severe Plasmodium falciparum malaria. Currently, PMC is given up to age two in perennial transmission settings. However, no recommendation exists for perennial settings with seasonal variation in transmission intensity, recently categorized as 'sub-perennial'.

A small molecule inhibitor of RNA-binding protein IGF2BP3 shows anti-leukemic activity

The RNA-binding protein IGF2BP3 is an oncofetal protein overexpressed in B-acute lymphoblastic leukemia and is critical for leukemogenesis in experimental models. With cancerspecific expression, functional dispensability for normal development, and an unleveraged prooncogenic function in mRNA homeostasis, IGF2BP3 represents an excellent target.

Ontogeny of plasma cytokine and chemokine concentrations across the first four months of human life in a Papua new Guinean cohort

Dynamic molecular changes in early life follow a robust ontogeny as the infant immune system adapts to the demands of its new environment. Studies of plasma immunomodulatory cytokines and chemokines have previously demonstrated ontogenetic patterns of immune development across the first week of life. However, how plasma cytokine and chemokines concentrations evolve over the first 4 months of life remains unknown. 

Minimising antibiotic use through prevention of childhood ear infections

Otitis media (OM), or middle ear infection, is one of the most common childhood illnesses globally. In Australia, OM remains a leading cause of antibiotic prescriptions in children, despite growing awareness of antimicrobial resistance (AMR) and the need for stewardship. Preventing OM not only reduces the burden of disease but also plays a critical role in curbing unnecessary antibiotic use and slowing the rise of AMR.

Understanding the Determinants of Use of the Australian Guide to Diagnosis of Fetal Alcohol Spectrum Disorder: Informing the Review, Development and Dissemination

The Australian Guide to the Diagnosis of fetal alcohol spectrum disorder (FASD), developed in 2016, is currently under review. This study aimed to understand how the Guide is used in practice and identify factors influencing its implementation.

Enhancing Anesthesia Research: The Imperative of Consumer Engagement Into Clinical Research

Britta Regli-von Ungern-Sternberg AM FAHMS MD, PhD, DEAA, FANZA Chair of Paediatric anaesthesia, University of Western Australia; Consultant