News & Events

Advanced Therapeutics: No longer science fiction – a look at how gene therapy is changing the game for children with SMA In our upcoming webinar, we will be visiting the topics of newborn bloodspot screen and genetic therapies in the context of treating Spinal Muscular Atrophy (SMA). We will hear from a panel of speakers who will discuss our centres success in establishing a multidisciplinary...

The Sydney Children’s Hospitals Network (SCHN) and Children’s Medical Research Institute (CMRI) are pleased to announce the establishment of the Australian Genome Therapeutics Centre (AGTC), which will transform the treatment of children with serious inherited diseases and contribute to the development of exciting new treatment options for a wide range of other diseases, including cancer, across...

Registration now open! Register now and join our 3rd webinar of the Advanced Therapeutics series. In this webinar, we will be focusing on Ocular Gene Therapy, the first in vivo gene therapy approved by the Therapeutic Goods Administration (TGA) here in Australia for clinical use in inherited retinal diseases. Inherited retinal diseases are a group of conditions that disproportionately affect c...

Background The 28 of February is International Rare Disease Day, an opportunity to raise awareness around rare diseases and improve access to treatment and medical representation for individuals with rare diseases and their families. There are over 300 million people living with one or more of over 6000 identified rare diseases around the world. Although individually uncommon, there ar...