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

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

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

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

Despite several calls for greater inclusion of pregnant people in non-obstetric clinical trials, their systematic exclusion remains common practice. Excluding pregnant individuals from clinical trials may result in unintended consequences such as inadequate treatment of medical conditions in pregnancy, inappropriate dosing of medications, and investigational therapies being used off-label outside of the context of a clinical trial, risking adverse events in the absence of demonstrated efficacy. ...

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

Rett syndrome is a pervasive neurological disorder with impaired gait as one criterion. ...

• 1. Telethon Kids Institute
• Our research
• Reports and Findings
• 2015
• July 2015

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. ...

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

This study investigated the capacity of three accelerometer-type devices to measure walking activity in Rett syndrome ...

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

The emergence of the novel severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), sparking a global pandemic, has driven an imperative to quickly design and conduct treatment studies. We strongly propose a national, coordinated approach for randomised controlled trials (RCTs) for coronavirus disease 2019 (COVID-19), future pandemics and inter-pandemic periods in Australia. ...

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