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Real-world benefits and tolerability of trofinetide for the treatment of Rett syndrome: The LOTUS studyAim: To describe the real-world effects of trofinetide in individuals with Rett syndrome (RTT) using the 18-month follow-up analysis of the LOTUS study.
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Pathways to Adolescent Pregnancy in Southeast Asia: Qualitative Evidence From Lived Experiences of Girls in Four CountriesHistorically, adolescent pregnancy has been conceptualized as an outcome of child marriage, but in Southeast Asia, the contexts and drivers of adolescent pregnancy are less well-understood. This study examines the relationship between adolescent pregnancy and child marriage and explores the drivers of pregnancy in Cambodia, Indonesia, Lao People's Democratic Republic and Malaysia among adolescents who have experienced pregnancy.
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Pain and recovery profiles following common orthopaedic surgeries in childrenLittle evidence exists on the postoperative trajectory after paediatric orthopaedic surgery. Pain and behavioural disturbance can have short- and long-term impacts on children and their families. An improved understanding of procedure-specific postoperative trajectories can enhance recovery. The primary outcome was to examine the duration and severity of postoperative pain experienced by children undergoing 10 commonly performed orthopaedic procedures.
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Automated Insulin Delivery Is Associated with Reduced Hospital Admissions and Costs for Acute Diabetes Complications in Children with Type 1 DiabetesThis study evaluated the association between insulin regimen, hospitalization for acute diabetes complications, and related health care costs in children with type 1 diabetes (T1D). Hospital admissions for diabetic ketoacidosis or hypoglycemia between January 5, 2022, and April 30, 2024, were analyzed in Western Australian children with T1D. Admissions due to newly diagnosed T1D were excluded. Incidence rate ratios were calculated using generalized estimating equations, adjusted for age, diabetes duration, and socioeconomic status.
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Psychometric evaluation of the Comprehensive Autistic Trait Inventory in autistic and non-autistic adultsMeasures of autistic traits are only useful – for pre-diagnostic screening, exploring individual differences, and gaining personal insight – if they efficiently and accurately assess autism as currently conceptualised while maintaining psychometric validity across different demographic groups. We recruited 1322 autistic and 1279 non-autistic adults who varied in autism status (non-autistic, diagnosed autistic, self-identifying autistic) and gender (cisgender men, cisgender women, gender diverse) to assess the psychometric properties of the Comprehensive Autistic Trait Inventory, a recently developed measure of autistic traits that examines six trait domains using 42 self-report statements.
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Assessing Clinical Deterioration in Children With Dark-Coloured Skin: A Scoping ReviewBackground: Signs of clinical deterioration may appear differently in children with dark-coloured skin. How to assess children in this cohort is currently poorly defined. Aim: To explore available information on the assessment of clinical deterioration in children with dark-coloured skin and identify research deficits.
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Evaluating the Introduction of Humidified High-Flow Nasal Cannula Therapy Into an Australian Aeromedical Service Within a Paediatric Population: A Retrospective Cohort StudyHumidified high flow (HHF) oxygen is increasingly used to treat acute respiratory illnesses in children; however, use during aeromedical transfer is not well described. This was a retrospective cohort study. Children who were transferred from rural locations and were initiated on HHF prior to transfer between 1 January 2015 and 31 December 2018 were identified from the Royal Flying Doctors Service database. Clinical variables prior to transfer, during flight and after transfer were collected from medical records and flight records.
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Amplitude-integrated electroencephalography compared with conventional video-electroencephalography for detection of neonatal seizuresBackground: Conventional video-electroencephalography (cEEG) is the reference standard for diagnosing and managing neonatal seizures. However, continuous bedside cEEG services are not available in most neonatal units. Hence, an alternative and relatively simple method called amplitude-integrated EEG (aEEG), which uses a limited number of scalp electrodes, has become popular. aEEG allows continuous bedside monitoring of the electrical activity of the brain in neonates.
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Tumor site-directed A1R expression enhances CAR T cell function and improves efficacy against solid tumorsCitation: Sek K, Chen AXY, Cole T, Armitage JD, Tong J, ……… Waithman J, Parish IA, et al. Tumor site-directed A1R expression enhances CAR T cell
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Parental Experiences of Having a Child Diagnosed With Septo-Optic DysplasiaSepto-optic dysplasia (SOD) is a congenital disorder affecting 1 in 10,000 births, defined by the presence of at least two of a clinical triad, consisting of optic nerve hypoplasia, midline brain defects and pituitary hormone deficiency. Children with SOD may have vision impairment, hormonal deficiencies, developmental disorders, or epilepsy, but the clinical picture is highly variable. The complexity of SOD, its interplay with family factors, and the need for multiple specialty commitments can make the diagnosis period a challenging time for families.