Read the latest information about our research program and global research developments.
Research News
Approaching Sanfilippo from the parent’s perspective
A study initiated by Cure Sanfilippo Foundation, USA, aimed to investigate the impact of Sanfilippo ...
Monday, 21st Jun 21
Research News
The world’s first zebrafish model of Sanfilippo: a game-ch...
Dr Jan Kaslin, from the Australian Regenerative Medicine Institute (ARMI), based at Monash Universit...
Tuesday, 15th Jun 21
Research News
Can measuring the brain help clinical trials for Sanfilippo?
Clinical trials for Sanfilippo syndrome rely on accurate & easy to measure biomarkers.
Researchers...
Thursday, 3rd Jun 21
Research News
Clues to lung symptoms in Sanfilippo
Researchers from Australia, Spain and the UK have studied the effect of Sanfilippo Syndrome on the l...
Tuesday, 25th May 21
Research News
New research on Sanfilippo type B enzyme replacement therapy
Research led by Prof. Patricia Dickson, based at The Lundquist Institute and Washington University h...
Monday, 10th May 21
Research News
New research shows high rates of hip problems in Sanfilippo ...
While there is a significant focus on neurodegenerative symptoms, it is important to recognise that ...
Thursday, 22nd Apr 21
Research News
Research sheds light on brain health of Sanfilippo carriers
Associate Professor Kim Hemsley and her team of researchers from South Australia have published rese...
Monday, 22nd Mar 21
Research News
Research update from the 2021 WORLDSymposium
The annual WORLDSymposium was held virtually this year due to the COVID-19 pandemic, but nevertheles...
Thursday, 4th Mar 21
Research News
Encouraging results from ongoing gene therapy clinical trial...
Abeona Therapeutics presented encouraging results from their ongoing gene therapy clinical trial for...
Monday, 15th Feb 21
Research News
Hopes for a new Sanfilippo type A clinical trial
Researchers at The University of North Carolina have developed a second-generation gene therapy for ...
Monday, 15th Feb 21
Research News
Research spotlight: ERT development for Sanfilippo type D
Towards the end of 2020, American researchers published an enzyme replacement therapy proof-of-conce...
Monday, 1st Feb 21
Research News
Speaker opportunity at virtual NORD conference
SHARE YOUR SANFILIPPO STORY INTERNATIONALLY … The National Organization for Rare Disorders (NORD)...
Wednesday, 13th Jan 21
Research News
First study on Sanfilippo caregiver preferences published
Our colleagues at the USA-based Cure Sanfilippo Foundation have published the first study of the pre...
Wednesday, 23rd Dec 20
Research News
Sanfilippo retinal study findings
Is the eye a window to the brain in Sanfilippo? The findings of a National Health and Medical Resea...
Wednesday, 16th Dec 20
Research News
Orchard Therapeutics reports early results in Sanfilippo cli...
Orchard Therapeutics has reported results from the first Sanfilippo type A patient treated in their ...
Thursday, 10th Dec 20
Research News
Congratulations, Andrew Shoubridge!
Andrew Shoubridge, the first recipient of the Sanfilippo Children’s Foundation top-up
scholarship...
Thursday, 10th Dec 20
Research News
3rd International Conference: Sanfilippo Syndrome & Related ...
Sanfilippo Children’s Foundation was proud to be a sponsor of the 3rd International Conference on ...
Thursday, 10th Dec 20
Research News
World Sanfilippo Awareness Day
November 16 is World Sanfilippo Awareness Day. Now in its second year, this international day aims t...
Thursday, 12th Nov 20
Research News
Sanfilippo clinical trial factsheets now available
The Foundation recently developed Sanfilippo clinical trial factsheets for families, which are avail...
Thursday, 8th Oct 20
Research News
Exciting Progress For Our Community
Exciting progress for our community and a new chapter for Meg Donnell and the Sanfilippo Children’...
Tuesday, 25th Aug 20
Research News
Introducing Dr. Jana Vukovic to our SAB
The Sanfilippo Children's Foundation is thrilled to be expanding our Scientific Advisory Board. Plea...
Wednesday, 22nd Jul 20
Research News
Targeting the immune systems as a treatment approach for San...
The Sanfilippo Children’s Foundation has awarded funds to Associate Professor Kim Hemsley (Flinder...
Wednesday, 8th Jul 20
Research News
New SAB member Assoc. Prof. Karin Borges
We're thrilled to announce another addition to our esteemed Scientific Advisory Board. Please join u...
Wednesday, 1st Jul 20
Research News
New SAB member Assoc. Professor Sarah Spencer
We're delighted to announce a new addition to our esteemed Scientific Advisory Board. Please join us...
Wednesday, 3rd Jun 20
Research News
Development of nanoparticle drug delivery for Sanfilippo
Dr. Maria Francisca Coutinho from Portugal’s National Institute of Health Dr. Ricardo Jorge (INSA)...
Thursday, 7th May 20
Research News
Orchard Therapeutics Update: First patient treated
Incredible news out of the UK with the announcement that the first Sanfilippo patient has been treat...
Wednesday, 29th Apr 20
Research News
PhD Student - Flinders University: Investigating biomarkers ...
The Sanfilippo Children’s Foundation is proud to be providing a supplementary scholarship to Leann...
Wednesday, 22nd Apr 20
Research News
COVID-19 Impact on Sanfilippo families and researchers
During this unprecedented time, we want to update you on how the COVID-19 situation is impacting chi...
Wednesday, 15th Apr 20
Research News
Digital Divides: Hacking the Human Genome (2SER All Things E...
LISTEN to this powerful 2SER All Things Equal podcast entitled 'Digital Divides: Hacking the Human G...
Thursday, 19th Mar 20
Research News
PhD Student Nazzmer Nazri
The Sanfilippo Children’s Foundation is proud to be providing a supplementary scholarship to Nazzm...
Monday, 9th Mar 20
Research News
Evaluating potential drug therapies for Sanfilippo
The Sanfilippo Children’s Foundation and Fondation Sanfilippo Suisse have awarded funds to Dr. Ade...
Monday, 9th Mar 20
Research News
Targeting Autophagy in Sanfilippo Syndrome
Louise O’Keefe from The University of Adelaide and the South Australian Health & Medical Research ...
Sunday, 8th Mar 20
Research News
Investigating the effects of Sanfilippo on the lungs
The Sanfilippo Children’s Foundation has awarded funds to Prof. Sandra Orgeig and Dr. Emma Parkins...
Monday, 2nd Mar 20
Research News
Rare disease in spotlight with National Strategic Action Pla...
The Hon Greg Hunt, Federal Minister for Health, has today announced $3.3 million to implement the fi...
Wednesday, 26th Feb 20
Research News
Biotechnology company, Allievex Corporation, enters licensin...
BioMarin has licenced its enzyme replacement therapy that is in clinical trial for Sanfilippo type B...
Friday, 25th Oct 19
Research News
Nature Journal: What to do when your grant is rejected
Our Foundation's research manager, Kristina Elvidge, is quoted in this article about the medical gra...
Friday, 25th Oct 19
Research News
New Abeona trial for older children
The original Abeona Sanfilippo Type A gene therapy trial (ABT-001) changed its criteria at the begin...
Wednesday, 18th Sep 19
Research News
Brain in a Dish: Adelaide's landmark study to cure child dem...
The Adelaide Advertiser: South Australian researchers will grow brain cells in a dish to fast-track ...
Monday, 26th Aug 19
Research News
VIDEO: Channel 10 Brain in a Dish project
Channel 10 National News Broadcast about the Sanfilippo Children's Foundation's $2.5 million 'Brain ...
Monday, 26th Aug 19
Research News
RESEARCH UPDATE: Study discovers best way to deliver gene th...
In 2016 the Sanfilippo Children’s Foundation awarded AUD$90,000 towards a project to improve deliv...
Monday, 26th Aug 19
Research News
$2.5 million for project to tackle Sanfilippo Syndrome
The Sanfilippo Children’s Foundation is thrilled to secure AUD$2 million of Government funding and...
Saturday, 24th Aug 19
Research News
The gene therapy revolution is here (Medical Xpress)
Gene therapy, for so long something that belonged to the future, has just hit the streets. Medical X...
Wednesday, 5th Jun 19
Research News
The gene therapy revolution is here. Medicine scrambling to ...
This article is an edited extract from Elizabeth Finkel’s address 'Gene therapy: cure but at what ...
Wednesday, 5th Jun 19
Research News
Collaborative Brain In A Dish Project to Search for New Trea...
Research Australia's journal 'Inspire' shares news of the Sanfilippo Children's Foundation's MRFF go...
Wednesday, 5th Jun 19
Research News
VIDEO: Seven News: Eugene Labs
Channel 7 News told the story of Matilda Page in a news story about the launch of an at-home, clinic...
Wednesday, 5th Jun 19
Research News
Eugene Labs launches home kit for parents to test for geneti...
Genetic and health start-up Eugene Labs has launched its at-home, clinical-grade genetic test servic...
Wednesday, 5th Jun 19
Research News
VIDEO: Future Proof: Genetic Screening (Channel 9 News)
Our Executive Director, Megan Donnell, shared her personal story with Channel 9 News yesterday for a...
Wednesday, 5th Jun 19
Research News
Research study: Is the eye a window to the brain for Sanfili...
Researchers in Adelaide have started an exciting research project to investigate changes in the eyes...
Wednesday, 5th Jun 19
Research News
SCF Advocacy award win
Megan Donnell, executive director of the Sanfilippo Children's Foundation, whose fight to progress r...
Wednesday, 5th Jun 19
Media Highlights
Chasing the Miracle (The Monthly)
"For Megan Donnell’s family, the DNA-altering revolution cannot come soon enough." Written by Scie...
Monday, 25th Feb 19
