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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.
Annual estimates of seasonal influenza vaccine effectiveness can guide global risk communication and vaccination strategies to mitigate influenza-associated illness. We aimed to evaluate vaccine effectiveness in countries using the 2023 southern hemisphere influenza vaccine formulation.
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.
Cerebral palsy (CP) should not be considered as a diagnosis but as a label; it is an umbrella term, primarily affecting posture and mobility. The definition is not sufficiently precise to guarantee agreement as to which patients to include under this label, but the additional inclusion criteria required are not yet internationally standardised.
Epidemiology of cerebral palsy (CP) aims to describe the frequency of the condition in a population and to monitor its changes over time, and a guide to the management of patients. Classification of CP is an important step toward describing more homogenous subgroups of persons with CP.
This study evaluated the clinical utility of the Parent Listening and Understanding Measure (PLUM) questionnaire as a potential screening tool for otitis media (OM) and associated hearing loss in Aboriginal and/or Torres Strait Islander and non-Aboriginal children.
Parents of children with physical/mental health and/or neurodevelopmental conditions often need to make disclosure decisions for their child. Disclosure can bring benefits (e.g., support) but can also risk harm (e.g., stigma). This systematic review aimed to consolidate research regarding parents' disclosure experiences to better understand how to support parents during this process.