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Showing results for "8"

Research

Tricho-hepatic-enteric syndrome (THES) without intractable diarrhoea

We report an 8 year old girl who was diagnosed with THES by the Undiagnosed Disease Program-WA with compound heterozygous pathogenic variants in SKIV2L

Research

Sex assigned at birth may modify health-related quality of life in children treated with peanut oral immunotherapy

The high burden of peanut allergy underscores the need for treatment options that improve patient health-related quality of life (HRQL). However, the modifying effect of sex assigned at birth on treatment-related outcomes remains poorly understood. We sought to investigate whether sex modifies treatment effect on the change in overall and subdomain HRQL during the PPOIT-003 trial.

Research

Edaravone for the Treatment of Motor Neurone Disease: A Critical Review of Approved and Alternative Formulations against a Proposed Quality Target Product Profile

Edaravone is one of two main drugs for treating motor neurone disease (MND). This review proposes a specific quality target product profile (QTPP) for edaravone following an appraisal of the issues accounting for the poor clinical uptake of the approved IV and oral liquid edaravone formulations. This is followed by a review of the alternative oral formulations of edaravone described in the published patent and journal literature against the QTPP.

Research

Kindy Moves: a protocol for establishing the feasibility of an activity-based intervention on goal attainment and motor capacity delivered within an interdisciplinary framework for preschool aged children with cerebral palsy

Preschool aged children with cerebral palsy (CP) and like conditions are at risk of performing below their peers in key skill areas of school readiness. Kindy Moves was developed to support school readiness in preschool aged children with CP and like conditions that are dependent on physical assistance and equipment throughout the day. The primary aims are to determine the feasibility of motor-based interventions that are functional and goal directed, adequately dosed and embedded into a play environment with interdisciplinary support to optimise goal-driven outcomes.

Research

The effect of azithromycin on structural lung disease in infants with cystic fibrosis (COMBAT CF): a phase 3, randomised, double-blind, placebo-controlled clinical trial

Structural lung disease and neutrophil-dominated airway inflammation is present from 3 months of age in children diagnosed with cystic fibrosis after newborn screening. We hypothesised that azithromycin, given three times weekly to infants with cystic fibrosis from diagnosis until age 36 months, would reduce the extent of structural lung disease as captured on chest CT scans.

News & Events

Recruits wanted for water immersion study

This University of Western Australia study, being conducted by a PhD student who is working with the Children's Diabetes Centre, aims to determine for the first time whether head out of water immersion or water-based exercise affects blood glucose levels in people with type 1 diabetes.

Research

Longitudinal analysis of respiratory outcomes among bauxite exposed workers in western Australia

This study investigated any associations between cumulative bauxite exposure and respiratory symptoms.

Research

Cyberbullying and the role of the law in Australian schools: Views of senior officials

Opinions of employees from the education and legal systems, regarding their perceptions of the role of the law and cyberbullying in Australian schools