Run your own race and find your cheerleaders along the way. That is the advice Melissa, mother of 18-year-old Lili, wants to share with the two million Australians impacted by rare diseases.

A new research project aimed at finding a cure for the genetic disorder Neurofibromatosis (NF) has received $350,000 in funding from the Flicker of Hope Foundation delivered through the Sydney Children’s Hospitals Foundation.

At four months of age Sophia is reaching all her developmental milestones. She demonstrates good head control while sitting supported and can even roll from her tummy to her back.

The benefits of a new precision medicine initiative, being piloted at Sydney Children’s Hospitals Network (SCHN), are already being seen in children and families with rare genetic diseases.

Earlier this year, several researchers from Kids Research, and our partners at the University of Sydney and UNSW, were awarded National Health and Medical Research Council (NHMRC) funding for their projects, which are focusing on a range of childhood diseases.

Professor Allison Tong, Co-Leader of Centre for Kidney Research Clinical Research group at Kids Research, Sydney Children’s Hospitals Network and Principal Research Fellow at the University of Sydney’s School of Public Health has been awarded the National Health and Medical Research Council (NHMRC) Elizabeth Blackburn Investigator Grant Award.

A gene therapy project to save infants’ lives has been named the top-ranked National Health and Medical Research Council (NHMRC) Ideas Grant for 2020.