Polgreen, Lynda E. http://orcid.org/0000-0002-2881-6138
Chen, Agnes H. http://orcid.org/0000-0003-4084-2838
Pak, Youngju
Luzzi, Anna
Morales Garval, Adolfo
Acevedo, Jonathan http://orcid.org/0000-0002-3012-5547
Bitan, Gal http://orcid.org/0000-0001-7046-3754
Iacovino, Michelina
O’Neill, Cara
Eisengart, Julie B. http://orcid.org/0000-0001-5340-017X
Funding for this research was provided by:
Cure Sanfilippo Foundation: https://curesanfilippofoundation.org/
Article History
Received: 6 December 2023
Accepted: 19 May 2024
First Online: 21 June 2024
Competing interests
: L.E.P. has research grants/contracts with BioMarin and Takeda; is a consultant for BioMarin, Lysogene and Denali Therapeutics; participated on a data safety monitoring board or advisory board for RegenxBio and Sangamo; and has received honoraria from BioMarin. A.H.C. has a research grant/contract with Takeda. J.A. and G.B. have research grants/contracts from the Cure Sanfilippo Foundation. C.O. is an employee of the Cure Sanfilippo Foundation, a non-profit organization; has served as a consultant for Denali Therapeutics, JCR Pharmaceuticals and Lysogene; and participated on a data safety monitoring board or advisory board for BioMarin, Lysogene and Ultragenyx. J.B.E. has research grants/contracts with Orchard Therapeutics, Prevail and Lysogene; received consulting fees from Denali Therapeutics, JCR Pharmaceuticals, Novel Pharma, Orchard Therapeutics, Regenxbio, Sanofi Genzyme, Sumitomo Pharma Co., Ltd. and Takeda; and received honoraria from Takeda. The remaining authors declare no competing interests.