Congratulations to the team from the Childhood Dementia Research Group (CDRG) at SAHMRI, who received the Translation and Impact Award at this year’s SAHMRI Awards night. Their project investigated a novel gene therapy-based treatment for the childhood-onset neurodegenerative disorder Sanfilippo syndrome, a rare genetic condition where the median age of death is 17 years and there is currently no approved treatment. As a result of the study, the CDRG’s Industry partner have initiated a multi-national Phase II/III human clinical trial of the therapy in children with Sanfilippo syndrome.
This is a fantastic achievement for one of our partner organisations, and a wonderful example of how the translational research being conducted in South Australia can have worldwide impact. We acknowledge that projects like this are a large team effort and we wish to recognise Group Leader Dr Kim Hemsley, and everyone else involved: Meghan Douglass, Sarah Tamang, Daniel Neumann, Barbara King, Helen Beard, Leanne Winner, Dr Adeline Lau, Dr Marten Snel, Dr Paul Trim, Dorota Gancarz (Quality Manager) and Heather Croucher (Legal Counsel).