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

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

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

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

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.

Registrations for the 2024 Solving Sanfilippo Symposium (31 May-1 June) are open. To see the program & register, visit events.humanitix.com/solving-sanfil…

In-person registration for the 2024 Solving Sanfilippo Symposium (31 May-1 June) closes 23 May. To see the full program & register, visit sanfilippo.org.au/research/solvi…

You can still register for online attendance at the 2024 Solving Sanfilippo Symposium (31 May-1 June). To see the program & register, visit sanfilippo.org.au/research/solvi…

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. lysosomaldiseasesummit.org

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: sanfilippo.org.au/blog/stem-cell… #MPSIII #ChildhoodDementia

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: ssl4.eir-parts.net/doc/4552/tdnet…

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: lysosomaldiseasesummit.org/agenda/

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: flindersfoundation.org.au/news/helper-dr…

Researchers at the UNC-Chapel Hill, 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: sanfilippo.org.au/blog/research-… #ChildhoodDementia #MPSIII

Consensus findings of a Feb 2024 Foundation for FDA 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: sanfilippo.org.au/blog/heparan-s…

🔬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.

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: sanfilippo.org.au/blog/research-… #ChildhoodDementia #MPSIII

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: lysosomaldiseasesummit.org/agenda/

🔬Project Update: Dr. Johannes Schlachetzki at UC San Diego & his team have completed their Sanfilippo CFoundtn & Fondation Sanfilippo Suisse Incubator project investigating microglia in #Sanfilippo. READ MORE: sanfilippo.org.au/research/funde… #ChildhoodDementia

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