Skip to content

Search

ORIGIN-1 trial: study protocol for an organoid-guided N-of-1 trial evaluating CFTR modulator response in cystic fibrosis with rare non-F508del mutations

Cystic fibrosis (CF) is a genetic condition caused by mutation of the cystic fibrosis transmembrane regulator (CFTR) gene. Recently licensed modulator therapies target the defective CFTR protein and have transformed the formerly life-limiting trajectory of people with CF (pwCF).

Global, regional, and national burden of meningitis, its risk factors, and aetiologies, 1990–2023: a systematic analysis for the Global Burden of Disease Study 2023

Meningitis remains the leading infectious cause of neurological disabilities globally, disproportionately affecting children younger than 5 years and populations in the African meningitis belt. Whereas previous global estimates focused on ten pathogen categories, this study presents the most comprehensive analysis to date, assessing the meningitis burden attributable to 17 causative pathogens based on the Global Burden of Diseases, Injuries, and Risk Factors Study (GBD) 2023 framework.

Modified Delphi Study to Establish Consensus on an Australasian Minimum Data Set for Pediatric Stroke Rehabilitation

This study aimed to develop a consensus-derived minimum data set for childhood stroke and establish an agreed set of tools to measure key functional outcomes identified as important by childhood stroke survivors and their families. 

Patterns of language and visuospatial lateralisation in three-year-old children

Little is known about how or when language and visuospatial processing lateralise in the brain, and if individual differences in lateralisation are related to early language or visuospatial abilities. We explored if patterns of language and visuospatial lateralisation are related to cognitive skills in young children.

Co-designing a new clinical pathway to support families with children identified as having early-stage type 1 diabetes in Western Australia

Children with early-stage (pre-symptomatic) type 1 diabetes are currently identified primarily via research-based screening programmes in Australia. Once identified, families live with the knowledge that their child has an increased chance of developing symptomatic, lifelong, insulin-requiring type 1 diabetes but have no specific clinical pathway available to them in Western Australia for accessing tailored support or education. This project aimed to co-design a new clinical pathway to address this unmet need.

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.

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.

Translating the Eczema Bathing Study: why context and infection burden matter

Asha Jacinta Bernadette Bowen Walton Ricciardo BA MBBS DCH FRACP PhD GAICD FAHMS OAM MBBS (hon) DCH FACD Head, Healthy Skin and ARF Prevention

Prevalence and Sociodemographic Variation of Allergic Diseases in Australia: Findings From the Australian National Health Survey

The prevalence of allergic diseases across the Australian population, in all regions and age groups, is not well documented. This study aimed to describe the prevalence and distribution of five allergic diseases (allergic rhinitis, asthma, drug allergy, eczema, and food allergy) and examine differences by sociodemographic factors. 

Psychometric validation of the quality of life Inventory − Disability (QI-Disability) among patients with Lennox-Gastaut syndrome and Dravet syndrome

To evaluate the psychometric properties of the Quality of Life Inventory -Disability (QI-Disability) for individuals with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), two rare developmental and epileptic encephalopathy conditions.