Neonatal sepsis is a leading cause of infant mortality worldwide with non-specific and varied presentation. We aimed to catalogue the current definitions of neonatal sepsis in published randomised controlled trials. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2021
• November 2021

This narrative review describes the evolution, range and relative strengths and weaknesses of endpoints used in late phase trials ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2019
• November 2019

Raelene Endersby R BSc (Hons) PhD Brainchild Fellow; Co-Head, Brain Tumour Research raelene.endersby@telethonkids.org.au Co-Head, Brain Tumour Research Raelene was awarded her PhD in 2003 from the Harry Perkins Institute for Medical Research (under ...

• 1. Telethon Kids Institute
• Projects

Nick Raelene Gottardo Endersby NG R MBChB FRACP PhD BSc (Hons) PhD Co-Head, Brain Tumour Research Brainchild Fellow; Co-Head, Brain Tumour Research nicholas.gottardo@telethonkids.org.au raelene.endersby@telethonkids.org.au Co-Head, Brain Tumour ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2023
• August 2023

Research on and commercial development of the artificial pancreas (AP) continue to progress rapidly, and the AP promises to become a part of clinical care. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2016
• January 2016

Despite initial improvements in survival of infants with ALL since establishment of the first pediatric cooperative group ALL trials, the poor outcome has... ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2014
• April 2014

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. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2021
• October 2021

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

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2011
• June 2011

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. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2022
• March 2022

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). ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2023
• January 2023

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

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2013
• June 2013

Convalescent plasma has been widely used to treat COVID-19 and is under investigation in numerous randomized clinical trials, but results are publicly available only for a small number of trials. The objective of this study was to assess the benefits of convalescent plasma treatment compared to placebo or no treatment and all-cause mortality in patients with COVID-19. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2021
• November 2021