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This study aims to describe the pattern and trends in acute rheumatic fever (ARF)/rheumatic heart disease (RHD)-related hospitalisations and costs for Australians aged <65 years.
CDKL5 deficiency disorder presents as a challenging condition with early-onset refractory seizures, severe developmental delays, and a range of other neurological symptoms. Our study aimed to explore the benefits and side effects of anti-seizure medications in managing seizures among individuals with CDKL5 deficiency disorder, drawing on data from the International CDKL5 Disorder Database.
Event-free survival considers other adverse events in addition to mortality. It therefore provides a more complete understanding of the effectiveness and consequences of treatment than standard survival measures, but is rarely reported at the population level for childhood cancer.
Individual-based models of infectious disease dynamics commonly use network structures to represent human interactions. Network structures can vary in complexity, from single-layered with homogeneous mixing to multi-layered with clustering and layer-specific contact weights. Here we assessed policy-relevant consequences of network choice by simulating different network structures within an established individual-based model of SARS-CoV-2 dynamics.
Valerie Verhasselt MD, PhD Head, Immunology and Breastfeeding 0402997617 Valerie.verhasselt@thekids.org.au Head, Immunology and Breastfeeding @
This article explores how relational approaches to policymaking across multiple levels and sectors of society might enable transformative climate justice. It draws on a unique case study from the state of Western Australia, where climate justice is thwarted by the coloniality of climate politics and the hegemony of the fossil fuel industry.
In response to critical gaps in medical oxygen access, 2 pressure swing adsorption (PSA) oxygen production centers were established using an ecosystem-strengthening strategy in Amhara, Ethiopia, in 2019. A qualitative study was conducted to assess enablers and bottlenecks to oxygen access at the hospital level after installation.
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 CDKL5 Clinical Severity Assessment is a comprehensive, content-validated measurement tool capturing the diverse challenges of cyclin-dependent kinase-like 5 deficiency disorder, a genetically caused developmental epileptic encephalopathy. The CCSA is divided into clinician-reported and caregiver-reported assessments. The aim of this study was to evaluate the factor structure of these measures through confirmatory factor analysis and evaluate their validity and reliability.
Copy number alterations (CNAs), resulting from the gain or loss of genetic material from as little as 50 base pairs or as big as entire chromosome(s), have been associated with many congenital diseases, de novo syndromes and cancer. It is established that CNAs disturb the dosage of genomic regions including enhancers/promoters, long non-coding RNA and gene(s) among others, ultimately leading to an altered balance of key cellular functions.