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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.

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.

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.

Overcoming challenges in measuring early childhood development across cultures

These challenges in measuring early childhood development are exacerbated when making comparisons across cultures

Complexities of conducting research in adolescent refugees resettling in Australia

Adolescent refugees resettling in Australia are a vulnerable and marginalised population and research to understand their health-care needs remains scarce

Relevance of Aboriginal Peer-Led Parent Support: Strengthening the Child Environment in Remote Areas

This research highlights the critical emerging role of peer support workers in home visiting family support in a remote area of Australia

Pre-service teachers: Knowledge, attitudes and their perceived skills in addressing student bullying

The lack of skills in managing future bullying and particularly cyberbullying incidents reported by pre-service teachers has social and emotional implications for young people

Cyber-Friendly Schools

This chapter describes a whole-school cyberbullying intervention developed and evaluated in Western Australia with secondary school students aged 13–18 years old

Is cellular heterogeneity merely a confounder to be removed from epigenome-wide association studies?

DNA methylation is a very sensitive measure of cell fate mix and therefore reveals much about underlying disease etiology including aspects of causation.

Environment Changes Genetic Effects on Respiratory Conditions and Allergic Phenotypes

Genotype-phenotype associations relating to bronchitis and allergy susceptibility are dependent on the environment