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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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Acceptability and Implementation Challenges of Benzathine Penicillin G Secondary Prophylaxis for Rheumatic Heart Disease in Ethiopia: A Qualitative StudyMonthly intramuscular injections of benzathine penicillin G (BPG) remain the cornerstone of secondary prophylaxis for acute rheumatic fever and rheumatic heart disease (RHD). The barriers to successful delivery of BPG may be patient- or service-delivery-dependent.
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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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Antibody responses against influenza A decline with successive years of annual influenza vaccinationInfluenza vaccine effectiveness and immunogenicity can be compromised with repeated vaccination. We assessed immunological markers in a cohort of healthcare workers (HCW) from six public hospitals around Australia during 2020-2021.
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Child and Family Characteristics Associated with Symptoms of Anxiety in Autistic Children: A Biobank StudyAutistic children have an increased likelihood of anxiety, but more research is needed on the characteristics that predict various types of anxiety in this population.
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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.
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The use of automated insulin delivery around physical activity and exercise in type 1 diabetes: a position statement of EASD and ISPADRegular physical activity and exercise (PA) are cornerstones of diabetes care for individuals with type 1 diabetes. In recent years, the availability of automated insulin delivery (AID) systems has improved the ability of people with type 1 diabetes to achieve the recommended glucose target ranges. PA provide additional health benefits but can cause glucose fluctuations, which challenges current AID systems.
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Biases in Routine Influenza Surveillance Indicators Used to Monitor Infection Incidence and Recommendations for ImprovementMonitoring how the incidence of influenza infections changes over time is important for quantifying the transmission dynamics and clinical severity of influenza. Infection incidence is difficult to measure directly, and hence, other quantities which are more amenable to surveillance are used to monitor trends in infection levels, with the implicit assumption that they correlate with infection incidence.