Sanfilippo Syndrome is a rare genetic condition that causes fatal brain damage. It’s referred to as a childhood disease as most patients never reach adulthood. Every year worldwide an estimated 2,000 babies are born with this neurodegenerative disease.
The mission of the Sanfilippo Children's Foundation is to fund medical research so a cure can be found in time for children battling Sanfilippo today and those born with it tomorrow.
Born seemingly healthy, over time the brain of children with Sanfilippo gets progressively damaged. Children experience hyperactivity, sleeplessness, dementia, loss of speech and cognitive skills, intellectual disability, cardiac issues, seizures, loss of mobility and finally death. There is currently no effective treatment or cure, and life expectancy is just 12-20 years.
You can read more about Sanfilippo and the Sanfilippo Children's Foundation by visiting the Foundation website.
Funds raised by the SF Super Series will go towards funding vital research projects that show potential to halt disease progression; repair damage caused by the condition; or improve the quality of life for children suffering from Sanfilippo.
Where will the funds go?
Funds raised in through this year's SF Super Series will directly fund three research projects as part of the Sanfilippo Children's Foundation's incubator program.
These projects will:
- Investigate targeting the immune system as a treatment approach for Sanfilippo at the South Australian Health & Medical Research Institute (SAHMRI)
- Search existing drugs that could be re-purposed for Sanfilippo at SAHMRI, Adelaide's Women's & Children's Hospital and University of Adelaide
- Improve the effectiveness of gene and enzyme replacement therapies at the Telethon Institute of Genetics & Medicine in Italy.
Read more about these exciting research projects here
Meet some of the Australian children battling Sanfilippo Syndrome and their families. These children are the reason why we do what we do! By joining us in the SF Super Series you are showing your dedication and commitment to progressing research for this catastrophic condition and bringing hope for these beautiful children.
Read the family stories