News

New study found fenfluramine was an effective and safe treatment for Dravet Syndrome, reducing the number of epileptic seizures by over 75 per cent. For Bronte, this meant she went from having 10 seizures a day to just one or two a week and even began having seizure free weeks.

The first ever clinical practice guidelines for children with Charcot-Marie-Tooth disease (CMT) have been published after a decade of work by an international team.

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.

Two young sisters with a blinding eye condition will now have access to ocular gene therapy after a new pathway to the treatment was discovered.

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.