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Cystic fibrosis (CF) lung disease commences early in the disease progression and is the most common cause of mortality.

CDKL5 Deficiency Disorder (CDD) is a rare genetic disorder with symptoms of epilepsy, developmental impairments, and other comorbidities. Currently, there are no outcome measures for CDD with comprehensive evidence of validation. This study aimed to evaluate the psychometric properties of the Quality of Life Inventory-Disability (QI-Disability) in CDD. Quality of Life Inventory-Disability was administered to 152 parent caregivers registered with the International CDKL5 Disorder Database (ICDD).

The lack of a consensus definition of neonatal sepsis and a core outcome set proves a substantial impediment to research that influences policy and practice relevant to key stakeholders, patients and parents.

The Opportunity This position provides the successful appointee the opportunity to work within an experienced clinical trial research team. They will

Although clinical trials are fundamental to advancing evidence-based practice, significant heterogeneity in outcome reporting poses a considerable challenge to the validity of systematic reviews. This inconsistency impedes the ability to compare, synthesise and interpret research findings effectively. In the field of paediatric airway management, this issue is particularly relevant because of the low incidence of critical events and the related high morbidity and mortality. The issue of inadequate and variable outcome reporting in clinical trials has been widely acknowledged, necessitating initiatives to enhance the quality of future research.

Gliomas account for nearly 30% of all primary central nervous system (CNS) tumors in children and adolescents and young adults (AYA), contributing to significant morbidity and mortality. The updated molecular classification of gliomas defines molecularly diverse subtypes with a spectrum of tumors associated with age-distinct incidence.

This review identifies challenges and barriers to successful development of drugs in neuro-oncology trials at the preclinical, clinical and translational stages that we believe has contributed to poor outcomes for patients over the last 30 years.

A standardised framework for selecting outcomes for evaluation in trials has been proposed by the Core Outcome Measures in Effectiveness Trials working group. However, this method does not specify how to ensure that the outcomes that are selected are causally related to the disease and the health intervention being studied. Causal network diagrams may help researchers identify outcomes that are both clinically meaningful and likely to be causally dependent on the intervention, and endpoints that are, in turn, causally dependent on those outcomes.

The recent announcement of the negative results of the TIGER- 2 phase 3 study of denufosol tetrasodium

Researchers developing a nasal therapy to prevent childhood ear infections and reduce overuse of antibiotics have received $300,000 in top-up funding.