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Radiographic and visual response to the type II RAF inhibitor tovorafenib in children with relapsed/refractory optic pathway glioma in the FIREFLY-1 trialDue to their anatomical locations, optic pathway gliomas (OPGs) can rarely be cured by resection. Given the importance of preserving visual function, we analyzed radiological and visual acuity (VA) outcomes for the type II RAF inhibitor tovorafenib in the OPG subgroup of the phase 2 FIREFLY-1 trial.
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“If you build it, they will come”: the convergence of funding, research and collaboration in paediatric brain cancer clinical trialsEach year, approximately 1000 children in Australia and New Zealand, aged 0–14 years, are diagnosed with cancer. Despite paediatric cancer accounting for less than 1% of all cancer cases, the impact on their families and communities is profound and disproportionate.
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A GWAS for grip strength in cohorts of children-Advantages of analysing young participants for this traitGrip strength is a proxy measure for muscular strength and a predictor for bone fracture risk among other diseases. Previous genome-wide association studies have been conducted in large cohorts of adults focusing on scores collected for the dominant hand, therefore increasing the likelihood of confounding effects by environmental factors.
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Protective factors, risk factors, and intervention strategies in the prevention and reduction of crime among adolescents and young adults aged 12–24 years: A scoping review protocolEvidence indicates that criminal behaviour in youth is linked with a range of negative physical, mental, and social health consequences. Despite a global decrease over the last 30 years, youth crime remains prevalent.
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Biomarkers of vaccine safety and efficacy in vulnerable populations: Lessons from the fourth international precision vaccines conferenceVaccination has been a cornerstone of public health, substantially reducing the global burden of infectious diseases, notably evident during the COVID-19 pandemic caused by SARS-CoV-2.
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Predictive gene expression signature diagnoses neonatal sepsis before clinical presentationNeonatal sepsis is a deadly disease with non-specific clinical signs, delaying diagnosis and treatment. There remains a need for early biomarkers to facilitate timely intervention. Our objective was to identify neonatal sepsis gene expression biomarkers that could predict sepsis at birth, prior to clinical presentation.
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The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmapIn recent years, a small number of people with rare diseases caused by unique genetic variants have been treated with therapies developed specifically for them. This pioneering field of genetic N-of-1 therapies is evolving rapidly, giving hope for the individualized treatment of people living with very rare diseases.
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Invasive group B streptococcal infections in Western Australia, 2000–2018Neonatal and puerperal sepsis are major manifestations of invasive group B streptococcal (Streptococcus agalactiae; iGBS) infections. International data indicate the importance of iGBS infections among non-pregnant adults.
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Development, construct validity and utility of a cross-culturally adapted Otitis Media-6 (OM-6) questionnaire for urban Aboriginal and/or Torres Strait Islander childrenTamara Chris Valerie Veselinovic Brennan-Jones Swift MClinAud PhD Clinical Research Fellow Head, Ear and Hearing Health Aboriginal Co-Director,
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Ecological-level factors associated with tuberculosis incidence and mortality: A systematic review and meta-analysisGlobally, tuberculosis (TB) is the leading infectious cause of morbidity and mortality, with the risk of infection affected by both individual and ecological-level factors. While systematic reviews on individual-level factors exist, there are currently limited studies examining ecological-level factors associated with TB incidence and mortality. This study was conducted to identify ecological factors associated with TB incidence and mortality.