Meet Ella McDonald, PhD Candidate at @‌Flinders University working in the @‌CedricBardyLab, who uses a ‘Brain in a Dish’ model of #Sanfilippo to test drugs that may improve quality of life for people affected & their families:bit.ly/3IxMUWz.

Project Update: Prof Kim Hemsley at @FlindersHmri, Prof Vito Ferro 🇮🇹🇦🇺 at UQ Chemistry and Molecular Biosciences & Drs Louise O’Keefe & Sher Li Tan Uni of Adelaide completed their Incubator project on purinergic signalling in #Sanfilippo funded by Sanfilippo CFoundtn. READ MORE: bit.ly/49qrWVq #ChildhoodDementia

Funded Project Update: Dr Maria Francisca Coutinho Maria Francisca Coutinho & her colleagues at Instituto Ricardo Jorge have completed their Incubator grant awarded from Sanfilippo CFoundtn to develop #SubstrateReduction therapy for #Sanfilippo . READ MORE: bit.ly/3uHPKVB #ChildhoodDementia

Australian researchers: Abstract submissions for those interested in presenting at the 2024 Solving Sanfilippo Symposium close 5 pm AEDT, 28 Feb. Template & more info available at bit.ly/3uui1ip. #MPSIII #ChildhoodDementia

Project Update: A/Prof Michael Lardelli from Uni of Adelaide & team have completed their Incubator project funded by Sanfilippo CFoundtn to develop 3 new zebrafish models of #Sanfilippo & undertake behavioural & transcriptomic analysis: bit.ly/3uoDpFM #ChildhoodDementia Michael Lardelli

Funded Project Update: Leanne Winner, Sanfilippo CFoundtn PhD top-up scholarship recipient at Flinders University Flinders UniversityHmri, has completed her project to discover new, easily accessible #biomarkers for #Sanfilippo . READ MORE:bit.ly/3wmg8of #ChildhoodDementia

Funded Project Update: A/Prof Coy Heldermon from UF Health Cancer Center & his colleagues have completed their Incubator grant awarded from Sanfilippo CFoundtn & Cure Sanfilippo Fdn to evaluate 2 types of #StemCells & their potential to treat #Sanfilippo : bit.ly/3uf1DCj #ChildhoodDementia

Our Head of Research, Lisa Melton is at #WORLDsymposium - the largest annual gathering of international researchers, clinicians, industry and patient orgs focused on lysosomal storage disorders. We look forward to bringing you updates on the latest research. Cure Sanfilippo Fdn

We are excited to announce SCF is a member of the International Sanfilippo Syndrome Alliance (ISSA), a global collaboration committed to finding a cure for Sanfilippo syndrome. Find out more about the alliance here: sanfilippoalliance.org

Funded Project Update: Prof. Andreas Schulze from The Hospital for Sick Children (SickKids) & team completed their Translational extension grant co-funded by Sanfilippo CFoundtn & Cure Sanfilippo Fdn to assess #SubstrateReduction drug candidates for #Sanfilippo . READ MORE: bit.ly/3vR0uAZ #ChildhoodDementia

Ewan Gerken is a PhD Candidate at The University of Adelaide who uses zebrafish models of Sanfilippo and early-onset Alzheimer’s Disease to study how these diseases affect the brain’s cells and zebrafish behaviour. Read more about his work: bit.ly/3QOt8cU

Today is World Sanfilippo Awareness Day! It's a great day to have a conversation with someone about Sanfilippo syndrome to help spread awareness. Find out more about how you can help at bit.ly/3QBApxZ

The International Symposium on MPS and Related Diseases will be held in Wurzburg, Germany from 4 to 7 April, 2024. Scientific & clinical program & a program for families and youth. Super-early bird registration until 15 November! Visit the website: mps2024.com

The Sanfilippo CFoundtn team were excited to be part of the Lysosomal Disease Summit, organised by Fabry, Australia, that brought together researchers, clinicians, patient advocates and industry reps to discuss research & care for this group of diseases. READ MORE: bit.ly/3MxQKRS

🩺 CLINICAL TRIAL NEWS: JCR Pharmaceuticals’ Phase I/II clinical trial of a new Enzyme Replacement Therapy, JR-441, has dosed the first patient with Sanfilippo type A in Germany. The therapy fuses sulfamidase with JCR’s BBB-penetrating technology. #MPSIII bit.ly/3u4uwRa

The Sanfilippo Children’s Foundation team are excited to be here at the inaugural Lysosomal Disease Summit in Melbourne. Looking forward to meeting more members of the clinical and research community. We have a booth at the expo, so come and say hi!

A pre-print article from an NIH-funded study by researchers at FLORIDA has found that without a cure, #Sanfilippo syndrome will lead to an estimated economic burden of US$1.55b over the next 20 years in the US. READ MORE: bit.ly/45sTKFP
#MPSIII #ChildhoodDementia

We’re excited that our Head of Research @‌DrLisaMelton is presenting at the Lysosomal Disease Summit 2023 hosted by @‌FabryAustralia, Melbourne 27-29 Oct. View the excellent program and register here: lysosomaldiseasesummit.org/agenda

TICKETS SELLING FAST FOR THE DECADENCE BALL IN SYDNEY ON 14 OCTOBER 2023

Enjoy superb food, wine and entertainment with host Tracey Spicer AM GAICD and help find a cure for the childhood dementia, Sanfilippo syndrome.

Don't miss out! Tickets available at aus.givergy.com/DecadenceBall2…

Thank you to our Family Ambassador Jillian O’Grady for sharing her family’s story at the SA branch @‌HGSAAustralasia Symposium on Friday. This advocacy and awareness raising ensures that exciting developments in genetic and genomic science will make an impact for #Sanfilippo