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Advancing Impactful Research for Adolescent Health and Wellbeing: Key Principles and Required Technical InvestmentsSubstantial progress in adolescent health research has been made over recent decades, but important knowledge gaps remain.
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Leaving no patient behind! Expert recommendation in the use of innovative technologies for diagnosing rare diseasesGenetic diagnosis plays a crucial role in rare diseases, particularly with the increasing availability of emerging and accessible treatments. The International Rare Diseases Research Consortium (IRDiRC) has set its primary goal as: "Ensuring that all patients who present with a suspected rare disease receive a diagnosis within one year if their disorder is documented in the medical literature".
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Notification of acute rheumatic fever and rheumatic heart disease in hospitalised people in the Midwest region of Western Australia, 2012–2022: a retrospective cohort studyAcute rheumatic fever and rheumatic heart disease are caused by untreated group A streptococcus infections. Their prevalence is much higher among First Nations people than other Australians.
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Association between Congenital Anomalies and Late-Onset Bacterial Infections in Neonates Admitted to Neonatal Intensive Care Units in Australia and New ZealandCompromised neonatal intensive care unit neonates are at risk of acquiring late-onset infections (late-onset sepsis [LOS]). Neonates born with congenital anomalies could have an additional LOS risk.
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Eating and exercise experiences of Australian trans and gender diverse folks: lived experience and stakeholder perspectivesTrans individuals face elevated health risks and socio-environmental challenges, influencing their engagement in health-protective behaviors (e.g. exercise and nutrition). Despite these challenges, there is a significant gap in understanding the specific eating and exercise experiences of Australian trans adults, including barriers to healthy behaviors and healthcare experiences. This study aims to address this gap by exploring these experiences, informing targeted interventions and healthcare practices to improve health outcomes.
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Global Lung Function Initiative reference values for multiple breath washout indicesMultiple breath washout is a lung function test based on tidal breathing that assesses lung volume and ventilation distribution. The aim of this analysis was to use the Global Lung Function Initiative methodology to develop all-age reference equations for the multiple breath washout indices lung clearance index and functional residual capacity.
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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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Comments and Controversies in Oncology: The Tribulations of Trials Developing ONC201Our international team highlights issues with efficacy reports in several studies on DMG with the new drug ONC201.
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Mental Health Problems Among Indonesian Adolescents: Findings of a Cross-Sectional Study Utilizing Validated Scales and Innovative Sampling MethodsThis study aimed to estimate the prevalence of mental health problems and identify potential risk and protective exposures for adolescents in Indonesia. An innovative sampling approach was applied to simultaneously recruit school- and community-based adolescents aged 16-18 years old from Jakarta (urban megacity) and South Sulawesi (remote province).
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Six months of hybrid closed-loop therapy improves diabetes-specific positive well-being, and reduces diabetes distress and fear of hypoglycemia: secondary analysis of a controlled trialThis analysis aimed to investigate diabetes-specific psychological outcomes among adults with type 1 diabetes (T1D) using hybrid closed-loop (HCL) versus standard therapy.