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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.
Children receiving treatment for acute myeloid leukemia (AML) are at high risk of invasive fungal disease (IFD). Evidence from pediatric studies support the efficacy of antifungal prophylaxis in reducing the burden of IFD in children receiving therapy for AML, yet existing antifungal agents have specific limitations and comparative data to inform the optimal prophylactic approach are lacking.
Bernadette Ricciardo MBBS (hon) DCH FACD PhD Candidate Bernadette.Ricciardo@thekids.org.au PhD Candidate Dr Bernadette Ricciardo is a PhD student on
Immunocompromised hosts experience more breakthrough infections and worse clinical outcomes following infection with COVID-19 than immunocompetent people. Prophylactic monoclonal antibody therapies can be challenging to access, and escape variants emerge rapidly. Immunity conferred through vaccination remains a central prevention strategy for COVID-19.
Bacille Calmette-Guérin (BCG) vaccine has immunomodulatory effects that may provide protection against unrelated infectious diseases. We aimed to determine whether BCG vaccination protects adults against COVID-19.
In Western Australia (WA), children aged 24 months living regionally or remotely (non-urban) have suboptimal vaccine uptake. As there has not yet been a systematic approach to understanding the facilitators and barriers to childhood vaccination in regional and remote WA, this study aimed to understand the views of key immunisation stakeholders regarding barriers and solutions.
Childhood infection might be associated with adverse child development and neurocognitive outcomes, but the results have been inconsistent.
Serosurveys are considered as a valuable tool in estimating population immunity and infection rates but recruitment of children to provide paediatric estimates can be challenging. A novel approach of sampling children undergoing anaesthesia was utilised for a SARS-CoV-2 serosurvey in Australian children and we explore the reasons for participation, feedback on the approach and importance of research into Coronavirus Diseases 2019 (COVID-19).
Older people with cognitive impairment are unrepresented in clinical research. Our objective was to review evidence for strategies to support their research inclusion and participation.
The underlying pathogenesis of pediatric obstructive sleep disordered breathing (SDB) and recurrent tonsillitis (RT) are poorly understood but need to be elucidated to develop less invasive treatment and prevention strategies.