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The Kids researchers who helped identify rare disorder now poised to help the hunt for treatment

Two The Kids Research Institute Australia researchers recognised for their role in building a global database for CDKL5 deficiency disorder are now helping to set the scene for clinical trials of much-needed potential treatments.

Research

Without training, they lacked knowledge. Without knowledge, they lacked confidence. Without confidence, they lacked victory (Julius Caesar)

Britta Regli-von Ungern-Sternberg MD, PhD, DEAA, FANZA Chair of Paediatric anaesthesia, University of Western Australia; Consultant Paediatric

Research

Anaesthesia related mortality in paediatrics is rare. There are limited data describing paediatric anaesthesia related mortality. This study determined the anaesthesia related mortality at a Tertiary Paediatric Hospital in Western Australia.

Research

Immunological mechanisms of vaccine-induced protection against COVID-19 in humans

Most COVID-19 vaccines are designed to elicit immune responses, ideally neutralizing antibodies (NAbs), against the SARS-CoV-2 spike protein. Several vaccines, including mRNA, adenoviral-vectored, protein subunit and whole-cell inactivated virus vaccines, have now reported efficacy in phase III trials and have received emergency approval in many countries.

People

Professor Christopher Blyth

Centre Head, Wesfarmers Centre of Vaccines and Infectious Diseases; Honorary and NHMRC Career Development Fellow, Paediatric Infectious Diseases Physician and Clinical Microbiologist

Research

A prospective cohort study assessing the reactogenicity of pertussis and influenza vaccines administered during pregnancy

Data on systemic and local reactions following receipt of TIV and dTpa during pregnancy support the safety of antenatal vaccination

Research

BEAT CF pulmonary exacerbations core protocol for evaluating the management of pulmonary exacerbations in people with cystic fibrosis

Cystic fibrosis (CF) is a rare, inherited, life-limiting condition predominantly affecting the lungs, for which there is no cure. The disease is characterized by recurrent pulmonary exacerbations (PEx), which are thought to drive progressive lung damage. Management of these episodes is complex and generally involves multiple interventions targeting different aspects of disease. The emergence of innovative trials and use of Bayesian statistical methods has created renewed opportunities for studying heterogeneous populations in rare diseases.

Research

Predicting the causative pathogen among children with osteomyelitis using Bayesian networks – improving antibiotic selection in clinical practice

We have demonstrated the potential use of Bayesian Networks in improving antibiotic selection for children with osteomyelitis

Research

Antecedent and persistent symptoms in COVID-19 and other respiratory illnesses: Insights from prospectively collected data in the BRACE trial

Some individuals have a persistence of symptoms following both COVID-19 (post-acute COVID-19 syndrome; PACS) and other viral infections. This study used prospectively collected data from an international trial to compare symptoms following COVID-19 and non-COVID-19 respiratory illness, to identify factors associated with the risk of PACS, and to explore symptom patterns before and after COVID-19 and non-COVID-19 respiratory illnesses.

Research

Consensus guidelines for optimising antifungal drug delivery and monitoring to avoid toxicity and improve outcomes in patients with haematological malignancy and haemopoietic stem cell transplant recipients, 2021

Antifungal agents can have complex dosing and the potential for drug interaction, both of which can lead to subtherapeutic antifungal drug concentrations and poorer clinical outcomes for patients with haematological malignancy and haemopoietic stem cell transplant recipients. Antifungal agents can also be associated with significant toxicities when drug concentrations are too high.