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BEAT-CF (Bayesian Evidence Adaptive Treatment for people with Cystic Fibrosis): description of a prospective cohort for nested studies in cystic fibrosis

Despite recent improvements in treatment modalities for cystic fibrosis (CF), there is currently limited evidence and a lack of consensus regarding optimal treatment strategies for the different aspects of CF, including pulmonary exacerbations (PEx). We aimed to establish a prospective cohort of people with CF (pwCF) to evaluate alternative approaches to managing CF in the era of modulator therapies.

Designing a paediatric teleotology pilot in the UK: Outcomes from a scoping review of the literature, with insights from the Ear Portal pilot service in Perth, Western Australia

To inform the design of a United Kingdom (UK) paediatric teleotology pilot by analysing global teleotology initiatives and drawing on insights from a successful paediatric teleotology pilot service in Perth, Western Australia (Ear Portal).

Psychosocial Outcomes in Parents of Children with Acute Lymphoblastic Leukaemia in Australia and New Zealand Through and Beyond Treatment

Parents of children with acute lymphoblastic leukaemia (ALL) experience emotional distress throughout their child's treatment course. This study describes the psychological experience of Australian and New Zealand parents of children diagnosed with ALL. 

A complete genome of an obligately lytic Pseudomonas aeruginosa bacteriophage, Minga-mokiny 4

We report the isolation of a bacteriophage with obligately lytic activity against Pseudomonas aeruginosa from wastewater. The reported phage, Minga-mokiny 4, appears to belong to the Schitoviridae family, is of the Litunavirus genus, and has a 72,362-bp genome. No known genes associated with lysogeny, bacterial resistance, or virulence were predicted.

Sex-specific white matter alterations in children exposed to high pregestational BMI

This study investigated whether exposure to high pregestational BMI (≥ 25 kg/m2) is associated with alterations in white matter microstructure in early childhood, explored sex-specific effects, and examined associations with cognitive performance.

Potential predictive value of CD8A and PGF protein expression in gastric cancer patients treated with neoadjuvant immunotherapy

Immunoneoadjuvant therapy has gained significant attention due to its remarkable advancements in cancer treatment. This study aimed to investigate the molecular mechanisms underlying immunoneoadjuvant therapy through a comprehensive multiomics analysis of samples from a registered clinical trial cohort.

Hospitalizations Following Complex Hip Surgery in Children with Intellectual Disability: A Self-Controlled Case Series Analysis

To evaluate the associations between complex hip surgery and subsequent hospitalizations in children with intellectual disability, including a subset of children with cerebral palsy.

International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA

The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.

Streptococcus pyogenes pharyngitis elicits diverse antibody responses to key vaccine antigens influenced by the imprint of past infections

Knowledge gaps regarding human immunity to Streptococcus pyogenes have impeded vaccine development. To address these gaps and evaluate vaccine candidates, we established a human challenge model of S. pyogenes pharyngitis. Here, we analyse antibody responses in serum and saliva against 19 antigens to identify characteristics distinguishing 19 participants who developed pharyngitis and 6 who did not.

Maternal Allergic Disease Phenotype and Infant Birth Season Influence the Human Milk Microbiome

Early infancy is a critical period for immune development. In addition to being the primary food source during early infancy, human milk also provides multiple bioactive components that shape the infant gut microbiome and immune system and provides a constant source of exposure to maternal microbiota. Given the potential interplay between allergic diseases and the human microbiome, this study aimed to characterise the milk microbiome of allergic mothers.