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Authors
Rocha, Júlio C. http://orcid.org/0000-0002-4977-8345
Hermida, Álvaro
Jones, Cheryl J.
Wu, Yunchou
Clague, Gillian E.
Rose, Sarah
Whitehall, Kaleigh B.
Ahring, Kirsten K.
Pessoa, André L. S.
Harding, Cary O.
Rohr, Fran
Inwood, Anita
Longo, Nicola
Muntau, Ania C.
Sivri, Serap
Maillot, François
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Funding
Funding for this research was provided by:
BioMarin Pharmaceutical
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Article History
Received: 13 October 2023
Accepted: 19 May 2024
First Online: 12 September 2024
Declarations
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: Not applicable.
: Not applicable.
: JCR has received consulting payments from Applied Pharma Research, BioMarin, Merck Serono, Nutricia, PTC Therapeutics, and Synlogic Therapeutics; speaker fees/payments from Applied Pharma Research, BioMarin, Cambrooke, LifeDiet, Merck Serono, Nutricia, PIAM, and Vitaflo; travel support from Applied Pharma Research, BioMarin, Glutamine, Merck Serono, PIAM; and research grants from BioMarin. AH has received consulting payments from Amicus Therapeutics, BioMarin, Chiesi, Genzyme, Shire, and Ultragenyx; speaker fees/payments from Alexion, Amicus Therapeutics, BioMarin, Genzyme, InMedica, Nutricia, Sobi, Takeda, and Vitaflo; travel support from Amicus Therapeutics, BioMarin, Chiesi, Genzyme, Inmedica, Sobi, and Vitaflo; and has participated as a clinical trial investigator for Ultragenyx. At the time this work was undertaken, CJJ and YW were employees of HCD Economics, a company sponsored by BioMarin to conduct this study and prepare the manuscript. GEC, SR, and KBW are employees and stockholders of BioMarin. KKA has received consulting payments from Arla Foods Ingredients, BioMarin, Homology, and Nutricia. ALSP has received speaker fees/payments from BioMarin. COH has received consulting and speaker fees/payments from BioMarin and has participated as a clinical trial investigator for BioMarin. FR is a managing partner of Met Ed who has received educational grants from BioMarin. AI has received consulting payments for and travel support to advisory boards from BioMarin. NL has received consulting payments for advisory boards from Alnylam, Amicus Therapeutics, Audentes/Astellas, BioMarin, BridgeBio/CoA Therapeutics, Chiesi/Protalix, Genzyme/Sanofi, HemoShear Therapeutics, Horizon Pharma, Jaguar Gene Therapy, Jnana Therapeutics, Leadiant Biosciences, Moderna, Nestlé Pharma, PTC Therapeutics, Recordati, Reneo, Takeda, and Ultragenyx; has received other consultancy payments from Synlogic Therapeutics; has received travel support from BioMarin; has participated as a clinical trial investigator for Aeglea, Amicus Therapeutics, Audentes/Astellas, AVROBIO, BioMarin, Chiesi/Protalix, Genzyme/Sanofi, HemoShear Therapeutics, Homology, Horizon Pharma, Moderna, Nestlé Pharma, Pfizer, PTC Therapeutics, Reneo, Synlogic Therapeutics, Takeda, Travere Therapeutics, and Ultragenyx; and has been Data Safety and Monitoring Chair for ACI Clinical. ACM has received consulting payments from Atheneum, Nestlé, and PTC Therapeutics; speaker fees/payments from AIM, Applied Pharma Research, and Nutricia; travel support from Nutricia; and has participated as a clinical trial investigator for Nutricia. SS has received speaker fees/payments from BioMarin and Sanofi. FM has received consulting payments from PTC Therapeutics and travel support from BioMarin.
