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Authors
Porter, Katherine Ackerman
O’Neill, Cara
Drake, Elise
Andrews, Sara M.
Delaney, Kathleen
Parker, Samantha
Escolar, Maria L.
Montgomery, Stacey
Moon, William
Worrall, Carolyn
Peay, Holly L. https://orcid.org/0000-0002-3053-7453
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Funding
Funding for this research was provided by:
cure sanfilippo foundation
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Article History
Received: 28 June 2021
Accepted: 4 April 2022
First Online: 25 April 2022
Declarations
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: The RTI International IRB exempted this study from IRB review, as the information obtained is recorded by the investigator in such a manner that the identity of the human participants can readily be ascertained, directly or through identifiers linked to the participants. The procedures used in this study adhere to the tenets of the Declaration of Helsinki.
: Written informed consent to participate and publish this data was obtained from all participants. No identifying information is included in the manuscript.
: Katherine Porter, Sara Andrews, and Holly Peay are employees of RTI International, which was contracted by Cure Sanfilippo Foundation to complete this work. Kathleen Delaney is an employee of BioMarin Pharmaceutical Inc. When this research was being conducted, Samantha Parker was the Chief Patient Access Officer at Lysogene, a biopharmaceutical company developing gene therapy for MPS IIIA. Maria Escolar is a consultant for two clinical trials. Cara O'Neill, Elise Drake, William Moon, Carolyn Worrall, and Stacey Montgomery report no competing interests exist.
