Adj. Associate Professor Paula Bray is the Network Director of Research for the Sydney Children’s Hospitals Network. Paula completed her PhD and post-doctoral studies in measurement science and improving clinical care through development and implementation of international clinical practice guidelines for Duchenne muscular dystrophy and Charcot-Marie-Tooth disease. Paula is committed to advancing healthcare delivery through unlocking value by implementing research-enabled care. She works closely with research leaders, clinicians and partners to achieve national and international research impact that works to solve the biggest challenges facing children’s health.

Dr Andrea Perez-Iturralde, Children’s Medical Research Institute, Scientist: Discusses the scientific innovation behind N=1 clinical trials, focusing on the design and pre-clinical implementation of such trials in paediatric advanced therapies. She offers insights into the challenges and opportunities in translating scientific research into clinical practice, emphasising the lab-based research that underpins these innovative approaches.

Adam Jaffe is the John Beveridge Professor and head of Paediatrics and Child Health at UNSW, Sydney, head of the Randwick Clinical Campus and a Respiratory Consultant at Sydney Children’s Hospital Randwick. Jaffe has expertise in rare diseases and chairs the Scientific and Medical Advisory Committee of Rare Voices Australia. He contributed to the writing of the National Strategic Action Plan for Rare Diseases and the National Recommendations for Rare Disease Health Care. Jaffe is also on the Rare Diseases International World Health Assembly resolution taskforce and the International Rare Disease Research Consortium n of 1 taskforce. Jaffe established the molecular and integrative precision medicine centre for cystic fibrosis at UNSW Sydney.

Dr Aung-Htut graduated with a PhD in Biochemistry and Molecular Genetics from the University of New South Wales (UNSW). Prior to her position as a senior research fellow (Co-Head of Molecular Therapy Laboratory) at the Centre for Molecular Medicine and Innovative Therapeutics (CMMIT), Murdoch University, she held postdoctoral positions at the UNSW and Centre for Cancer Biology, University of South Australia working on cellular aging and transcriptional regulation of programmed cell death, respectively. Her current research focuses on designing and evaluating therapeutic antisense oligonucleotides for various diseases, including multiple sclerosis, juvenile-onset Parkinson’s, and Pompe disease. Dr. Aung-Htut aims to broaden the application of these therapeutic oligonucleotides to treat both rare genetic disorders and cardiovascular disease, the leading cause of death. Currently, Dr Aung-Htut and colleagues are driving towards innovative precision therapies for children with rare diseases, specifically n-of-1 approaches.

Since 2017 Sarah has Chaired the Sydney Children’s Hospital Network (SCHN) Human Research Ethics Committee (HREC), a Lead HREC under the National Mutual Acceptance Scheme and the NSW Health’s Statewide Early Phase Clinical Trial HREC for paediatrics. Sarah is an Associate Professor, Children's Hospital Westmead Clinical School, University of Sydney. Her research focuses on developing evidence-based interventions to prevent and treat childhood obesity and diabetes and she is currently on the scientific advisory panel of the EDIT Collaboration which brings together clinicians, researchers and individuals with lived experience from around the world to improve treatment for people affected by obesity and eating disorders.

Ms. Kris Pierce (MHealthSc (HealthProm&HealthEd), MWellness, Nursing) is a distinguished leader in consumer involvement and advocacy within the healthcare sector. As the Director of Consumer Involvement at Child Unlimited, UNSW, she has developed and implemented consumer engagement frameworks and led patient-centred initiatives. She has co-founded and leads SCN2A Australia and has contributed to the International League Against Epilepsy (ILAE), Global Genes, and The Epilepsy Foundation. Recognised through her induction into the Victorian Women’s Honour Roll and a nomination for the Children’s Healthcare Australasia Medal of Distinction, she continues to enhance patient and family engagement, ensuring consumer voices are integral to healthcare decision-making. Most recently, Kris was appointed to Medical Services Advisory Committee – Evaluation Sub-Committee.