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Preclinical efficacy of azacitidine and venetoclax for infant KMT2A-rearranged acute lymphoblastic leukemia reveals a new therapeutic strategyInfants with KMT2A-rearranged B-cell acute lymphoblastic leukemia (ALL) have a dismal prognosis. Survival outcomes have remained static in recent decades despite treatment intensification and novel therapies are urgently required.
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Early markers of adult symptoms of depression and anxiety in the Raine StudyEarly intervention offers the potential to mitigate adult mental illness; however, trials spanning decades present significant challenges, necessitating predictive early markers useable in trial settings. We hypothesised that parent evaluation using the child behaviour checklist (CBCL) total problem score at age two years predicted adult depressive and anxious symptoms and explored other potential parent ratings.
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Human Milk Sodium and Potassium as Markers of Mastitis in Mothers of Preterm InfantsThis prospective longitudinal study examined changes in milk sodium concentration (Na) and sodium:potassium ratio (Na:K), microbiological culture, milk production, and breast health in relation to mastitis after preterm birth.
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Living closer to the beach is associated with better socioemotional development in young boysNatural outdoor environments such as green and blue spaces have increasingly been seen as key health and wellbeing determinants for adults. However, it is unclear if these effects are seen in young children. We examined the associations between access to natural green and blue space and young children's socioemotional development.
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Potential immunological effects of gender-affirming hormone therapy in transgender people – an unexplored area of researchThere are well-described sex-based differences in how the immune system operates. In particular, cisgender (cis) females have a more easily activated immune system; associated with an increased prevalence of autoimmune diseases and adverse events following vaccinations. Conversely, cis males have a higher threshold for immune activation, and are more prone to certain infectious diseases, such as coronavirus disease (COVID-19).
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Short-Term Diabetic Retinopathy Status in People with Type 1 Diabetes Commencing Automated Insulin DeliveryRapid improvements in glucose control may lead to early worsening of diabetic retinopathy (EWDR). There is a need to demonstrate safety in people commencing automated insulin delivery (AID) due to the known efficacy in rapid glycemic improvement. We aimed to investigate short-term DR outcomes in people (aged ≥13 years) with type 1 diabetes after initiation of AID (use ≥6 months).
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Mapping national, regional and local prevalence of hypertension and diabetes in Ethiopia using geospatial analysisThis study aimed to map the national, regional and local prevalence of hypertension and diabetes in Ethiopia.
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Eating and drinking abilities and respiratory and oral health in children and young adults with cerebral palsyTo investigate the potential risk factors of respiratory illness (ethnicity, oral health, and eating and drinking ability) in children and young adults with cerebral palsy.
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The Use of Interdisciplinary Approaches to Understand the Biology of Campylobacter jejuniCampylobacter jejuni is a bacterial pathogen recognised as a major cause of foodborne illness worldwide. While Campylobacter jejuni generally does not grow outside its host, it can survive outside of the host long enough to pose a health concern. This review presents an up-to-date description and evaluation of biological, mathematical, and statistical approaches used to understand the behaviour of this foodborne pathogen and suggests future avenues which can be explored.
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Longitudinal Study of Indigenous Children: Adolescent never smoking and associations with individual, social, and environmental factorsFuelled by the tobacco industry, commercial tobacco use is a major cause of preventable morbidity and mortality among Aboriginal and Torres Strait Islander peoples. Preventing adolescent smoking initiation is critical to reducing uptake. Understanding individual, social, and environmental factors that are protective against smoking can inform prevention strategies.
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The overlapping burden of the three leading causes of disability and death in sub-Saharan African childrenDespite substantial declines since 2000, lower respiratory infections (LRIs), diarrhoeal diseases, and malaria remain among the leading causes of nonfatal and fatal disease burden for children under 5 years of age (under 5), primarily in sub-Saharan Africa.
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X-Linked Hypophosphataemia and Burosumab: A Systemic Disease With a New TreatmentX linked hypophosphataemia (XLH) is a systemic, chronic condition that significantly impairs quality of life. In XLH, a phosphate regulating endopeptidase homologue X-linked (PHEX) gene mutation leads to excess fibroblast growth factor 23 (FGF23), causing hypophosphataemia and subsequent rickets, lower limb deformity, pain and other sequelae, however there are likely other non-FGF23 mediated mechanisms contributing to disease
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Improving disease surveillance data analysis, interpretation, and use at the district level in TanzaniaAn effective disease surveillance system is critical for early detection and response to disease epidemics. This study aimed to assess the capacity to manage and utilize disease surveillance data and implement an intervention to improve data analysis and use at the district level in Tanzania.
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Characterizing the Nature of Alexithymia in Autistic Adults: Validation of the Perth Alexithymia QuestionnaireAlexithymia—a trait characterized by difficulties in emotion processing—is of high interest in the autism field. However, the lack of validated alexithymia measures for autistic individuals limits progress. This study aimed to address this gap by examining the psychometric properties of the Perth Alexithymia Questionnaire (PAQ) across autistic and non-autistic samples. Using the PAQ, we investigated how alexithymia manifests in autistic individuals and its links with poor mental health outcomes (anxiety).
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The COVID-19 Pandemic Affects Seasonality, With Increasing Cases of New-Onset Type 1 Diabetes in Children, From the Worldwide SWEET RegistryTo analyze whether the coronavirus disease 2019 (COVID-19) pandemic increased the number of cases or impacted seasonality of new-onset type 1 diabetes (T1D) in large pediatric diabetes centers globally.
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Caregiver broader autism phenotype does not moderate the effect of early caregiver-mediated support on infant language outcomesCaregiver-mediated supports in general have shown mixed evidence for enhancing language outcomes in infants at higher likelihood of autism. While caregivers play a substantial role in caregiver-mediated supports, little is known about whether caregivers' own subclinical autistic features - known as broader autism phenotype - may moderate infant language outcomes.
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Lifting the curtains of silence: Patient perceptions towards needs and responsibilities in contributing to the prevention of healthcare-associated infections and antimicrobial resistanceBeyond the use of policy and system-focused approaches, it has been established globally that patients can play a role in enhancing the health care landscape. However, efforts to meaningfully translate patient engagement strategies that promote participation by hospitalized patients in relevant infection prevention and antimicrobial stewardship activities have not yet been realized. This study mapped the key factors acting as barriers and facilitators of patient engagement using a theoretical framework to identify potential new approaches to promote engagement.
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Caregiver-reported meaningful change in functional domains for individuals with developmental and epileptic encephalopathy: A convergent mixed-methods designTo investigate how caregivers of children with developmental and epileptic encephalopathy and severe developmental impairments describe meaningful change for functional domains and why it is important.