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Research

Real-world benefits and tolerability of trofinetide for the treatment of Rett syndrome: The LOTUS study

Aim: 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.

Research

Pain and recovery profiles following common orthopaedic surgeries in children

Little 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.

Research

Automated Insulin Delivery Is Associated with Reduced Hospital Admissions and Costs for Acute Diabetes Complications in Children with Type 1 Diabetes

This 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.

Research

Intranasal phage therapy overcomes antibody neutralization challenges in pulmonary Pseudomonas aeruginosa infections

Phage therapy is a promising approach against multidrug-resistant infections, yet systemic administration can lead to incomplete cures. We investigated the distribution, immune responses, and efficacy of the therapeutic phage KPP10 delivered via intranasal or intraperitoneal routes in murine Pseudomonas aeruginosa lung infection models.

Research

Finding the optimal regimen for Mycobacteroides abscessus treatment (FORMaT) in people with Mycobacteroides abscessus pulmonary disease

Mycobacteroides abscessus (MABS) is within the non-tuberculous mycobacteria family. It inhabits soil and water, exhibits multi-antibiotic resistance and causes opportunistic lung infections, which may progress to symptomatic MABS-pulmonary disease (MABS-PD) associated with substantial morbidity, increased healthcare utilisation, impaired quality of life and increased mortality. 

Research

Evaluating the Introduction of Humidified High-Flow Nasal Cannula Therapy Into an Australian Aeromedical Service Within a Paediatric Population: A Retrospective Cohort Study

Humidified 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. 

Research

Intrauterine growth restriction promotes hypothalamic circadian dysregulation in adult mouse offspring

Adverse prenatal conditions can induce intrauterine growth restriction and increase the risk of adulthood metabolic disease. Mechanisms underlying developmentally programmed metabolic disease remain unclear but may involve disrupted postnatal circadian rhythms and kisspeptin signalling. 

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Examining the overlap in lymphatic filariasis prevalence and malaria insecticide-treated net access-use in endemic Africa

Eradication and elimination strategies for lymphatic filariasis (LF) primarily rely on multiple rounds of annual mass drug administration (MDA), but also may benefit from vector control interventions conducted by malaria vector control programs. We aim to examine the overlap in LF prevalence and malaria vector control to identify potential gaps in program coverage. 

Research

Parental Experiences of Having a Child Diagnosed With Septo-Optic Dysplasia

Septo-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.

Research

Global participatory wastewater surveillance to understand mpox clade diversity in war and conflict-affected countries

War and conflict severely disrupt public health systems, compromising infectious disease surveillance in many affected regions. Mpox, a re-emerging zoonotic disease, poses a growing global threat, especially in areas where traditional monitoring is inaccessible.The mpox virus has distinct clades with varying transmission and severity.