Wondering why landmarks in Australia are lighting up purple tonight? Raising awareness of Sanfilippo syndrome and our amazing famiiles around the world 💜 #worldsanfilippoday

🔬Project Update: Dr. Johannes Schlachetzki at @UCSD & his team have completed their @SFCFoundtn & Fondation Sanfilippo Suisse Incubator project investigating microglia in #Sanfilippo. READ MORE: https://t.co/WrSLTuQjJ2 #ChildhoodDementia

Don’t forget to register for the 2024 Lysosomal Disease Summit to be held in Melbourne, 18-20 October, bringing together researchers & clinicians from Australasia. View the program & register:

Australian researchers have compared how 2 gene therapy delivery routes impact the #retina in #Sanfilippo. While IV injected therapy led to better outcomes in the retina, combo therapy/multiple delivery methods may be needed: https://t.co/d0zcF3gu8r #ChildhoodDementia #MPSIII

🔬Update: A/Prof Alessandro Fraldi & team have completed their project on the anti-amyloid drug CLR01 in Sanfilippo, finding it has potential in multiple subtypes; earlier administration is more effective & can improve outcomes if delivered with gene therapy.

Consensus findings of a Feb 2024 @reaganudall Fdn/@FDA workshop on CSF HS as a surrogate biomarker for neuropathic MPS have been published. This crucial work opened the door to accelerated approvals for #Sanfilippo therapies. Read more:

Researchers at the @UNC, USA, and its Gene Therapy Center have published findings exploring the potential of a new gene therapy for #Sanfilippo type C that uses #ExtracellularVesicles to cross-correct cells: https://t.co/YfuyBpOyX3 #ChildhoodDementia #MPSIII

We’re delighted to see Prof Hemsley and Dr Lau have received Flinders Foundation funding to pursue combination therapy with chaperone drugs developed with support from SCF and others:

Registration and preliminary program are available for the 2nd Annual Lysosomal Disease Summit in Melbourne, Australia, 18-20 October 2024. Additional speakers & topics to be announced soon. View speakers here:

JCR Pharma and MEDIPAL have received the green light from the Japanese regulatory authority to commence a trial of their enzyme replacement therapy for Sanfilippo syndrome type B, which will be delivered via an intravenous injection. Read more:

Researchers in Canada & the USA have found stem cell transplantation in a #Sanfilippo type C mouse model reduces neuroinflammation and improves some behavioural symptoms. READ MORE: https://t.co/X36vFabCOf #MPSIII #ChildhoodDementia

Abstract Submission for the 2nd Annual Lysosomal Disease Summit in Melbourne, Australia are closing on 14 June 2024! The summit, presented by Fabry Australia, will be held 18-20 October 2024. Speaker scholarship applications are also available. https://t.co/FnHnW0SmEw

You can still register for online attendance at the 2024 Solving Sanfilippo Symposium (31 May-1 June). To see the program & register, visit

In-person registration for the 2024 Solving Sanfilippo Symposium (31 May-1 June) closes 23 May. To see the full program & register, visit

Registrations for the 2024 Solving Sanfilippo Symposium (31 May-1 June) are open. To see the program & register, visit

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: https://t.co/ht0nLqKBgl.

Project Update: Prof Kim Hemsley at @FlindersHmri, Prof @VitoFerro8 at @UQ_SCMB & Drs Louise O’Keefe & Sher Li Tan @UniofAdelaide completed their Incubator project on purinergic signalling in #Sanfilippo funded by @SFCFoundtn. READ MORE: https://t.co/9dU1xOpzA7 #ChildhoodDementia

Funded Project Update: Dr Maria Francisca Coutinho @lysogirl & her colleagues at @irj_pt have completed their Incubator grant awarded from @SFCFoundtn to develop #SubstrateReduction therapy for #Sanfilippo. READ MORE: https://t.co/Q8tfQ8VqPZ #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 https://t.co/E2FUDrdJGh. #MPSIII #ChildhoodDementia

Project Update: A/Prof Michael Lardelli from @UniofAdelaide & team have completed their Incubator project funded by @SFCFoundtn to develop 3 new zebrafish models of #Sanfilippo & undertake behavioural & transcriptomic analysis: https://t.co/PC76B26v2Z #ChildhoodDementia @ADandCD