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Authors
Stepien, Karolina M.
Burton, Barbara K.
Bober, Michael B.
Campeau, Philippe M.
Ellaway, Carolyn
Bhattacharya, Kaustuv
Guffon, Nathalie
Hinds, David
Hunt, Abigail
Lail, Alice
Lin, Shuan-Pei
Magner, Martin
Murphy, Elaine
Reisewitz, Pascal
Mitchell, John J.
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Funding
Funding for this research was provided by:
BioMarin Pharmaceutical (BioMarin Pharmaceutical)
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Article History
Received: 30 April 2025
Accepted: 23 September 2025
First Online: 30 October 2025
Declarations
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: The MARS study protocol was reviewed and approved by all applicable institutional review boards and ethics committees for participating study sites. All participants gave written informed consent, or, in the case of individuals aged < 18 years, written consent was obtained from a legally authorized representative. All publications related to the MARS registry have been overseen by a dedicated Scientific Publication Committee.
: Not applicable.
: KMS is a Principal Investigator in MARS for BioMarin Pharmaceutical Inc. BKB has received consulting payments from Aeglea, Agios, Altrna, Aro, BioMarin Pharmaceutical Inc., JCR Pharma, Jnana, Maze, Moderna, Passage Bio, PTC Therapeutics, Takeda, and Travere; has participated as a clinical trial investigator for BioMarin Pharmaceutical Inc., Denali, JCR Pharma, Sangamo, and Ultragenyx; and has received speaker fees/payments from BioMarin Pharmaceutical Inc., Chiesi, Horizon, Takeda, and Ultragenyx. MBB is an employee and owner of Tyra Biosciences; and has received consulting payments from and participated as a clinical trial investigator for BioMarin Pharmaceutical Inc. PMC has provided consultation/advisory services for BioMarin Pharmaceutical Inc., Ipsen, and Sanofi. CE has received honorarium for participation in a MARS Medical Advisory Board from BioMarin Pharmaceutical Inc. KB has nothing to report. NG has received consulting payments and travel support from and participated as a clinical trial investigator in MARS for BioMarin Pharmaceutical Inc.; is a Principal Investigator in clinical trials for Chiesi, JCR Pharma, Sanofi, Takeda, and Ultragenyx; and has received consulting/speaker fees from Chiesi, JCR Pharma, Sanofi, and Ultragenyx. DH, AH, and PR are employees of and may hold stock ownership and shareholdings in BioMarin Pharmaceutical Inc. AL has nothing to report. S-PL has provided advisory board services to BioMarin Pharmaceutical Inc. MM has received lecture honoraria and consultancy fees from BioMarin Pharmaceutical Inc., Chiesi, and Takeda. EM has participated as a clinical trial investigator for BioMarin Pharmaceutical Inc. JJM has received consulting payments from BioMarin Pharmaceutical Inc., Denali, Sanofi-Genzyme, Takeda, and Ultragenyx; has received research grants from BioMarin Pharmaceutical Inc., Sanofi-Genzyme, and Takeda; and has participated as a clinical trial investigator in MARS for BioMarin Pharmaceutical Inc.
