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The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.
There is currently limited data regarding the physical activity behaviors of trans and gender diverse people (including binary and non-binary identities; henceforth trans). The aim of this review was to synthesize the existing literature in this area, with a focus on physical activity behaviors as they relate to health (e.g. health benefits, risks of adverse health outcomes).
Platinum-based chemotherapy in combination with anti-PD-L1 antibodies has shown promising results in mesothelioma. However, the immunological mechanisms underlying its efficacy are not well understood and there are no predictive biomarkers to guide treatment decisions.
Andrew Videos Whitehouse Watch and listen to Andrew PhD Deputy Director (Research); Angela Wright Bennett Professor of Autism Research at The Kids
For individuals living with rare neurodevelopmental disorders, particularly those who are at the most severe end of the spectrum, standardized outcome measures may lack the sensitivity to capture small but meaningful changes.
Despite impacting mankind since ancient times, tuberculosis (TB) persists as the leading cause of death from an infectious disease. TB can remain latent and further research is required to understand activation risk and the risks vs. the benefits of treating latent infection. Drug resistance poses an escalating threat to treating active disease and achieving cure.
Dementia is a health priority for Indigenous peoples. Here, we reviewed studies on the prevalence of dementia or cognitive impairment among Indigenous populations from countries with a very high Human Development Index (≥0·8). Quality was assessed using the Joanna Briggs Institute risk-of-bias tool and CONSolIDated critERia for strengthening the reporting of health research involving Indigenous peoples (CONSIDER), with oversight provided by an Indigenous Advisory Board.
We present lung virome data recovered through shotgun metagenomics in bronchoalveolar lavage fluid from an infant with cystic fibrosis, who tested positive for Stenotrophomonas maltophilia infection. Using a bioinformatic pipeline for virus characterization in shotgun metagenomic data, we identified five viral contigs representing Pseudomonas phages classified as Caudoviricetes.
Drug-resistant tuberculosis (DR-TB) remains a major public health challenge in China, with varying treatment outcomes across different regions. Understanding the spatial distribution of DR-TB treatment outcomes is crucial for targeted interventions to improve treatment success in high-burden areas such as Hunan Province. This study aimed to map the spatial distribution of DR-TB treatment outcomes at a local level and identify sociodemographic and environmental factors associated with poor treatment outcomes in Hunan Province, China.
As COVID-19 vaccinations rolled out globally from late 2020, rules and recommendations regarding vaccine use in pregnancy shifted rapidly. Pre-registration COVID-19 vaccine trials excluded those who were pregnant. Initial Australian medical advice did not routinely recommend COVID-19 vaccines in pregnancy, due to limited safety data and little perceived risk of local transmission.