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Rett syndrome (RTT) is a severe neurodevelopmental disorder affecting predominantly females and associated with variants in the MECP2 gene. Recent success in clinical trials have resulted in an expanded use of the Rett Syndrome Behaviour Questionnaire (RSBQ) for clinical and research purposes.
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.
Regular exercise can be beneficial for people living with a severe mental illness. By better understanding the perspectives and challenges of adults with severe mental illness who are engaged in exercise, we can enhance the design and implementation of exercise programs to better support their mental health and recovery.
In mid-2018, the Australian childhood 13-valent pneumococcal conjugate vaccine schedule changed from 3+0 to 2+1, moving the third dose to 12 months of age, to address increasing breakthrough cases of invasive pneumococcal disease (IPD), predominantly in children aged >12 months. This study assessed the impact of this change using national IPD surveillance data.
Standardised psychometric measures are used in mental health care and research settings to identify risk, assist diagnosis, and assess symptom severity. Standardised scoring of these measures involves transforming respondents' raw scores using binary sex norms. However, scoring manuals offer no guidance as to appropriate scoring methods for trans and non-binary respondents.
To describe Aboriginal and Torres Strait Islander communities' processes, positioning and experiences of health and medical research and their recommendations.
Despite increasing urbanisation, little is known about skin health for urban-living Aboriginal children and young people (CYP, aged <18 years). This study aimed to investigate the primary care burden and clinical characteristics of skin conditions in this cohort.
To explore parents' experiences of using continuous glucose monitoring in their young children with early-stage type 1 diabetes, being followed in the Australian Environmental Determinants of Islet Autoimmunity (ENDIA) study.
Although social factors and culture are significant determinants of health in Aboriginal and Torres Strait Islander peoples, little is known academically about key interpersonal and social experiences of this population during the perinatal period, or how early attachments are formed through culture. This study addressed this gap in the literature.
Medicine acceptability is crucial for paediatric drug development, yet its assessment remains challenging due to the multifaceted nature of sensory attributes like taste, smell, and mouthfeel. Traditional methods of acceptability evaluation often involve complex questionnaires and lack standardisation, leading to difficulties in a comparative analysis across studies.