Mazzachi, Paris https://orcid.org/0009-0006-5974-7901
McDonald, Ella https://orcid.org/0009-0006-0852-0723
Greenberg, Zarina https://orcid.org/0000-0002-0103-8321
Puerta, Alejandra Noreña https://orcid.org/0009-0001-9531-1180
Tran, Jenne
De Silva, Manam Inushi https://orcid.org/0000-0002-5517-5903
Christensen, Cade https://orcid.org/0000-0001-5661-3787
Adams, Robert https://orcid.org/0000-0001-6254-0385
Loskarn, Sebastian https://orcid.org/0009-0002-4984-5963
Beard, Helen
Zabolocki, Michael https://orcid.org/0000-0002-1856-5523
Elmasri, Meera
Maack, Megan https://orcid.org/0000-0003-0870-0338
Elvidge, Kristina L. https://orcid.org/0000-0002-9763-344X
Hutchinson, Mark R. https://orcid.org/0000-0003-2154-5950
O’Neill, Cara https://orcid.org/0000-0002-9377-259X
Hemsley, Kim M.
Melton, Lisa https://orcid.org/0000-0002-6314-359X
Smith, Nicholas https://orcid.org/0000-0003-2409-9239
Bardy, Cedric https://orcid.org/0000-0001-8321-0852
Funding for this research was provided by:
Department of Health | National Health and Medical Research Council (MRFF MRF2024419)
Department of Education and Training | Australian Research Council (FT180100565)
Article History
Received: 12 September 2024
Accepted: 13 March 2026
First Online: 7 April 2026
Competing interests
: M.M. is the founder and Executive Director of the Sanfilippo Children’s Foundation and is the founder, Director and CEO of the Childhood Dementia Initiative. N.S. and K.H. serve on the advisory boards of the Sanfilippo Children’s Foundation and the Childhood Dementia Initiative. K.H. was also the Chair of the US National MPS Society Scientific Advisory Board. K.E. is Head of Scientific Affairs of the Childhood Dementia Initiative. L.M. was Head of Research at the Sanfilippo Children’s Foundation. C.O. is the Chief Science Officer of the Cure Sanfilippo Foundation and serves on advisory boards for the Ultragenyx Gene Therapy Advisory Committee and the NINDS Ultra-rare Gene-based Therapy External Consultant Board. C.B. is an inventor on the BrainPhys patent (US Patent App. 18/133,237). The remaining authors declare no competing interests.