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The translation gap between knowledge production and implementation into clinical practice and policy is an ongoing challenge facing researchers, funders, clinicians and policy makers globally. Research generated close to practice and in collaboration with end users is an approach that is recognised as an effective strategy to facilitate an improvement in the relevance and use of health research as well as building research capacity amongst end users.
Pathogenic variants in the CDKL5 gene result in CDKL5 deficiency disorder (CDD), which is characterized by early-onset epilepsy, severe developmental delay, and often, cortical visual impairment. Validated clinical outcome measures are needed for future clinical trials to be successful. This study aimed to adapt the Rett Syndrome Hand Function Scale for CDKL5 deficiency disorder and evaluate its feasibility, acceptability, content validity, and reliability.
Pineoblastoma is a rare brain tumor usually diagnosed in children. Given its rarity, no pineoblastoma-specific trials have been conducted. Studies have included pineoblastoma accruing for other embryonal tumors over the past 30 years.
There is growing evidence that lung function in early-life predicts later lung function. Adverse events over the lifespan might influence an individual’s lung function trajectory, resulting in poor respiratory health. The aim of this study is to identify early-life risk factors and their impact on lung function trajectories to prevent long-term lung impairments.
Caregivers play a crucial role in supporting the development of their children's emotion regulation. This study validated the Parental Assistance with Child Emotion Regulation (PACER) Questionnaire in a sample of 491 caregivers of young children ≤ 5 years.
The availability of age-appropriate, taste-masked oral solid medications for the paediatric population is currently inadequate. We have developed a novel chocolate-based drug delivery platform to taste-mask bitter drugs commonly utilised in the hospital setting, but there is limited evidence regarding parent's perspectives on these medications.
With immune checkpoint therapy (ICT) having reshaped the treatment of many cancers, the next frontier is to identify and develop novel combination therapies to improve efficacy. Previously, we and others identified beneficial immunological effects of the vitamin A derivative tretinoin on anti-tumour immunity.
Socio-economic spatial patterning of fast-food outlets can result in disparities in the availability and access of food across geographic areas, contributing to health inequalities. This study investigated whether area-level socio-economic disparities exist in fast-food availability across the Perth metropolitan region of Western Australia.
Current exercise guidelines for individuals with type 1 diabetes (T1D) do not consider the impact that high altitude may have on blood glucose levels (BGL) during exercise.
The aim of this narrative review is to summarise efficacy and pharmacokinetic data for Plasmodium vivax in children. The burden of P. vivax malaria in children continues to remain a significant public health issue, and the need for improved treatment regimens for this vulnerable population is critical.