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Benefits, implementation and sustainability of innovative paediatric models of care for children with type 1 diabetes: a systematic reviewThe evidence about the acceptability and effectiveness of innovative paediatric models of care for Type 1 diabetes is limited. To address this gap, we synthesised literature on implemented models of care, model components, outcomes, and determinants of implementation and sustainability.
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Mapping fertility rates at national, sub-national, and local levels in Ethiopia between 2000 and 2019Fertility rates are key indicators of population health and demographic change, influencing economic development, healthcare planning, and social policies. Understanding subnational variation in fertility rate is important for effective geographical targeting and policy prioritization. This study aimed to identify geographic variation, trends, and determinants of fertility rates in Ethiopia over the past two decades.
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Too far from care? A descriptive analysis of young Australian mental health aeromedical retrievalsYoung Australians living in rural and remote locations have poorer mental health outcomes and higher rates of self-harm and suicide than their major city counterparts. Significant service gaps and barriers exist in accessing general and youth-specific mental health services. With a lack of access, comes delays in treatment and associated poorer outcomes. This paper describes the characteristics of young people requiring an aeromedical retrieval (AR) for acute inpatient psychiatric care.
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Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trialCDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Ganaxolone, a neuroactive steroid, reduces the frequency of major motor seizures in children with CDD. This analysis explored the effect of ganaxolone on non-seizure outcomes.
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Prevalence of tuberculosis infection among contacts of drug-resistant tuberculosis patients: A systematic review and meta-analysisContact investigations with drug-susceptible tuberculosis (DS-TB) patients have demonstrated a high prevalence of tuberculosis infection (TBI). However, the prevalence of TBI among individuals in close contact with drug-resistant tuberculosis (DR-TB) patients is poorly understood. This systematic review and meta-analysis aimed to determine the prevalence of TBI among household and non-household contacts of DR-TB patients.
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Patient-advocate-led global coalition adapting fit-for-purpose outcomes measures to assure meaningful inclusion of DEEs in clinical trialsExisting clinical tools that measure non-seizure outcomes lack the range and granularity needed to capture skills in developmental and epileptic encephalopathy (DEE)-affected individuals who also fall in the severe to profound range of intellectual disability. This effectively excludes those with severe impairments from clinical trials, impeding the ability of sponsors to evaluate disease-modifying therapies.
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Efficacy thresholds and target populations for antiviral COVID-19 treatments to save lives and costs: a modelling studyIn 2023 severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) was declared endemic, yet hospital admissions have persisted and risen within populations at high and moderate risk of developing severe disease, which include those of older age, and those with co-morbidities. Antiviral treatments, currently only available for high-risk individuals, play an important role in preventing severe disease and hospitalisation within this subpopulation.
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Seasonal malaria chemoprevention and the spread of Plasmodium falciparum quintuple-mutant parasites resistant to sulfadoxine–pyrimethamine: a modelling studySeasonal malaria chemoprevention (SMC) with sulfadoxine-pyrimethamine plus amodiaquine prevents millions of clinical malaria cases in children younger than 5 years in Africa's Sahel region. However, Plasmodium falciparum parasites partially resistant to sulfadoxine-pyrimethamine (with quintuple mutations) potentially threaten the protective effectiveness of SMC. We evaluated the spread of quintuple-mutant parasites and the clinical consequences.
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The ETO2 transcriptional cofactor maintains acute leukemia by driving a MYB/EP300-dependent stemness programTranscriptional cofactors of the ETO family are recurrent fusion partners in acute leukemia. We characterized the ETO2 regulome by integrating transcriptomic and chromatin binding analyses in human erythroleukemia xenografts and controlled ETO2 depletion models. We demonstrate that beyond its well-established repressive activity, ETO2 directly activates transcription of MYB, among other genes.
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Interorganelle phospholipid communication, a house not so dividedThe presence of membrane-bound organelles with specific functions is one of the main hallmarks of eukaryotic cells. Organelle membranes are composed of specific lipids that govern their function and interorganelle communication. Discoveries in cell biology using imaging and omic technologies have revealed the mechanisms that drive membrane remodeling, organelle contact sites, and metabolite exchange.