Read the latest information about our research program and global research developments.
Research News
Prof Kim Hemsley and Dr Adeline Lau: Celebrating 20 years wo...
Twenty years ago, Kim Hemsley and Adeline Lau began their work at the Women’s and Children’s Hos...
Wednesday, 17th Aug 22
Research News
SOBI Sanfilippo type A enzyme replacement trial results publ...
Results from a clinical trial to evaluate the safety and effectiveness of SOBI003, a modified sulfam...
Thursday, 4th Aug 22
Research News
Chaperone therapy shows promise in Sanfilippo type C mouse m...
In work partly funded by Sanfilippo Children’s Foundation, a team of researchers, led by Professor...
Monday, 18th Jul 22
Research News
From misdiagnosis to early diagnosis: can we rewrite the sto...
Improving early diagnosis would remove the long, distressing diagnostic odyssey for families. It wou...
Monday, 27th Jun 22
Research News
State of play: Development of therapies targeting neuroinfla...
Neuroinflammation is inflammation that affects the brain or spinal cord. Researchers looking at Sanf...
Wednesday, 8th Jun 22
Research News
The importance of caregivers’ perspectives when evaluating...
A recent study has collected data from caregivers of patients with Sanfilippo to support clinical ou...
Tuesday, 17th May 22
Research News
Clinical trial updates from WORLDSymposium
The annual international Lysosomal Storage Diseases conference WORLDSymposium was held in San Diego ...
Wednesday, 20th Apr 22
Research News
A study of Fluoxetine in mice with Sanfilippo type A
Researchers from TIGEM, Federico II University, and IBBC in Italy, have studied a molecule called Fl...
Wednesday, 30th Mar 22
Research News
Brain in a Dish - project update
The Brain in a Dish research project began in July 2019 with the support of a $2 million grant from ...
Monday, 14th Mar 22
Research News
Exploring gene activity to understand symptoms and severity ...
Researchers have sought to get a better understanding of the biology that lies behind the variation ...
Monday, 7th Mar 22
Research News
The impacts of COVID-19-related distance learning on childre...
The COVID-19 pandemic has affected children’s education, with many classrooms moved online and tea...
Wednesday, 23rd Feb 22
Research News
Prestigious Dementia Australia Fellowship for young Sanfilip...
In what is a fantastic outcome, both for a talented young researcher and for Sanfilippo syndrome res...
Wednesday, 16th Feb 22
Research News
Proudly launching the Global Roadmap For Sanfilippo Syndrome...
Sanfilippo Children’s Foundation (Australia), Cure Sanfilippo Foundation (USA) and our sister orga...
Wednesday, 9th Feb 22
Research News
New type A ERT candidate on track for clinical trial
Japanese company, JCR Pharmaceuticals, has received an orphan drug designation from the European Com...
Tuesday, 1st Feb 22
Research News
New mouse model helps researchers see into the Sanfilippo ty...
Recently, researchers in the USA, in collaboration with biotech company Phoenix Nest Inc, developed ...
Friday, 21st Jan 22
Research News
Supporting patients with Sanfilippo using music
A study led by researchers at the University of Deusto in Spain has described three patients with Sa...
Wednesday, 5th Jan 22
Research News
Profile a Sanfilippo Researcher: Dr. Elvira De Leonibus
We are very grateful to all the researchers around the world working tirelessly to solve Sanfilippo....
Wednesday, 8th Dec 21
Research News
USA team awarded funding to study ultra-rare Sanfilippo type...
USA-based biotech company, Phoenix Nest Biotech, has been awarded a US$3.4 million grant from the US...
Wednesday, 1st Dec 21
Research News
Feeding and gut problems in Sanfilippo
While Sanfilippo Syndrome predominantly affects the brain, it also has an impact on many other body...
Friday, 26th Nov 21
Research News
A new Sanfilippo brain cell model provides clues on brain dy...
An Australian team of researchers has developed a human Sanfilippo type A neural cell model and used...
Thursday, 18th Nov 21
Research News
State of play: Development of enzyme replacement therapies f...
There are currently no approved treatment options for Sanfilippo. Several therapeutic avenues are be...
Tuesday, 9th Nov 21
Research News
Long-term data from a Type A Enzyme Replacement Therapy tria...
Pharmaceutical company Shire, subsequently acquired by Takeda in 2019, sponsored two ERT clinical tr...
Wednesday, 27th Oct 21
Research News
Profile a Sanfilippo Researcher: Dr. Maria Francisca Coutinh...
Meet Dr. Maria Francisca Coutinho, a researcher at the National Institute of Health Dr. Ricardo Jorg...
Friday, 15th Oct 21
Research News
State of play: Development of gene therapies for Sanfilippo
There are currently no approved treatment options for Sanfilippo. Several therapeutic avenues are be...
Thursday, 7th Oct 21
Research News
Type B gene therapy trial releases long-term follow-up resul...
Results from a 5.5-year follow-up study have been released for a completed Sanfilippo type B gene th...
Friday, 17th Sep 21
Research News
Lysosomal storage disorders - 1 in 5,000
Sanfilippo Syndrome belongs to a group of around 50 diseases collectively named lysosomal storage di...
Monday, 13th Sep 21
Research News
Coming at the heparan sulfate problem from the outside
New research has found that clearing the heparan sulfate outside of cells in Sanfilippo can reduce d...
Friday, 27th Aug 21
Research News
MPS2021 Conference Summary
MPS2021, the 16th International Symposium on mucopolysaccharidoses and related disorders, was held v...
Friday, 20th Aug 21
Research News
Profile of a Sanfilippo Researcher: A/Prof Vito Ferro
Meet Associate Professor Vito Ferro, a chemist at The University of Queensland.
Wednesday, 18th Aug 21
Research News
State of play: Development of substrate reduction therapies ...
There is no approved cure or therapy for Sanfilippo, but Substrate Reduction Therapy, or SRT, is one...
Friday, 13th Aug 21
Research News
State of play: Development of chaperone therapies for Sanfil...
There are currently no approved treatment options for Sanfilippo Syndrome, but several therapeutic a...
Thursday, 5th Aug 21
Research News
Research helps explain autism-like symptoms in Sanfilippo
Research published in the prestigious journal Nature Communications has helped reveal what causes so...
Wednesday, 21st Jul 21
Research News
Brain signalling in early-stage Sanfilippo: new insights fro...
A study led by Professor Alexey Pshezhetsky (University of Montreal) has investigated the role of sy...
Wednesday, 14th Jul 21
Research News
A Spotlight on Attenuated Sanfilippo
Research into attenuated forms of Sanfilippo Syndrome has been given a boost with recent funding to ...
Thursday, 8th Jul 21
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
