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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

Pathways to Adolescent Pregnancy in Southeast Asia: Qualitative Evidence From Lived Experiences of Girls in Four Countries

Historically, 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.

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. 

Research

“That's not fair on my kid”: Carers' perspectives on sport participation and experiences for children in out-of-home care

Children in out-of-home care participate in less organised sport than children from other household structures, potentially reducing opportunities for improvements in social, developmental, and health outcomes. Despite this, little is known about barriers and facilitators of sport participation for children in care. We aimed to explore carers' perspectives on the influences on children in care's participation and experiences in organised sport.

Research

Low-intensity parent- and clinician-delivered support for young autistic children in Aotearoa New Zealand: a randomised controlled trial

Aotearoa New Zealand does not provide publicly-funded intensive autism support. While parent-mediated supports are promising, children and families may also benefit from direct clinician support. We tested the efficacy of a low-intensity programme involving parent- and clinician-delivered support for autistic children.

Research

Associations between mental health profiles and later school outcomes

The dual-factor model of mental health proposes that high wellbeing and low distress are necessary to define mental health. This study used latent profile analysis to identify mental health profiles in a sample of 3,587 Australian grade 6 students and explored the association between mental health profiles and school outcomes measured in grades 7 and 9.

Research

Disruption of cotranscriptional splicing suggests that RBM39 is a therapeutic target in acute lymphoblastic leukemia

There are few options for patients with relapse/refractory B-cell acute lymphoblastic leukemia, thus this is a major area of unmet medical need. Here, we reveal that inclusion of a poison exon in RBM39, which could be induced both by CDK9 or CDK9 independent CMGC (cyclin-dependent kinases, mitogen-activated protein kinases, glycogen synthase kinases, CDC-like kinases) kinase inhibition, is recognized by the nonsense-mediated mRNA decay pathway for degradation.