Whitehall, Kaleigh B. http://orcid.org/0009-0009-9090-3580
Rose, Sarah
Clague, Gillian E.
Ahring, Kirsten K.
Bilder, Deborah A.
Harding, Cary O.
Hermida, Álvaro
Inwood, Anita
Longo, Nicola
Maillot, François
Muntau, Ania C.
Pessoa, André L. S.
Rocha, Júlio C.
Rohr, Fran
Sivri, Serap
Said, Jack
Oshinbolu, Sheun
Sibbring, Gillian C.
Funding for this research was provided by:
BioMarin Pharmaceutical Inc.
Article History
Received: 25 April 2023
Accepted: 5 May 2024
First Online: 12 August 2024
Declarations
:
: Not applicable.
: Not applicable.
: KBW, SR, and GEC are employees and stockholders of BioMarin. KKA has received consulting payments from Arla Foods Ingredients, BioMarin, Homology, and Nutricia. DAB has received consulting payments from BioMarin, Encoded Therapeutics, Synlogic Therapeutics, and Taysha Gene Therapies, and travel support from BioMarin. COH has received consulting and speaker fees/payments from BioMarin and has participated as a clinical trial investigator for 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. 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 and 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, Takeda, Travere Therapeutics, and Ultragenyx; and has been Data Safety and Monitoring Chair for ACI Clinical. FM has received consulting payments from PTC Therapeutics and travel support from BioMarin. ACM has participated as a clinical trial investigator for Nutricia; has received consulting payments from Atheneum, Nestlé, and PTC Therapeutics; speaker fees/payments from AIM, Applied Pharma Research, and Nutricia; and travel support from Nutricia. ALSP has received speaker fees/payments from BioMarin. JCR has received consulting payments from Applied Pharma Research, BioMarin, Merck Serono, Nutricia, PTC Therapeutics, and Synlogic, and speaker fees/payments from Applied Pharma Research, BioMarin, Cambrooke, LifeDiet, Merck Serono, Nutricia, PIAM, and Vitaflo, as well as travel support from Applied Pharma Research, BioMarin, Glutamine, Merck Serono, PIAM, and research grants from BioMarin. FR is a managing partner of Met Ed who has received educational grants from BioMarin. SS has received speaker fees/payments from BioMarin and Sanofi. GCS is an employee of Prime Access. JS and SO were employees of Prime Access at the time the study was undertaken. Prime Access (a division of Prime, Knutsford, UK) is a company sponsored by BioMarin to conduct this study and prepare the manuscript.