Klein, T. L. http://orcid.org/0000-0002-7154-4342
Bender, J.
Bolton, S.
Collin-Histed, T.
Daher, A.
De Baere, L.
Dong, D.
Hopkin, J.
Johnson, J.
Lai, T.
Pavlou, M.
Schaller, T.
Žnidar, I.
Funding for this research was provided by:
Sanofi
Article History
Received: 2 February 2024
Accepted: 16 June 2024
First Online: 10 July 2024
Declarations
:
: All patients provided informed consent to participate in the Registries.
: Not applicable.
: TK received honoraria for meeting attendance and advisory boards from Sanofi. TK is President and CEO of National MPS Society. JB is an employee of Sanofi. SB received travel grants from Amicus Therapeutics, Sangamo Therapeutics and Biomarin, and received travel grants and honorarium from Sanofi. TCH is the CEO of IGA, with which she participated in advisory boards for Avrobio. IGA received grants from Sanofi, Prevail, Pfizer, Avrobio, Takeda, M6P and Gain. IGA received consulting fees from UK Gaucher Association and Gaucher Outcome Survey. TCH received payment for expert testimony from NICE. AD received grants and royalties from Casa Hunter, Federação Brasileira das Associações de Doenças Raras (FEBRARARAS) and Casa dos Raros; consulting fees, speaking honoraria, payment for expert testimony, meeting attendance support, advisory board from Casa Hunter; meeting support attendance from FEBRARARAS, and patents issued with Casa dos Raros. DD is an employee of Sanofi and is a member of the PCORI Rare Disease Advisory Panel. JH participated in advisory boards for Zevra Therapeutics, Azafaros and the International Niemann-Pick Disease Registry; board member of Uplifting Athletes and National Niemann-Pick Disease. JJ is an employee and executive director of FSIG; FSIG received grants from 4D Molecular Therapeutics, Amicus Therapeutics, Avrobio, Chiesi Global Rare Diseases, Codexis, Freeline Therapeutics, Idorsia Pharmaceuticals, Sangamo Therapeutics, Sanofi, Siglion Therapeutics and Spark Therapeutics; received honoraria from Chiesi Global Rare Diseases, Idorsia Pharmaceuticals, Sanofi, Spark Therapeutics, Takeda and Protalix; received meeting attendance support from Chiesi Global Rare Diseases and Sanofi. JJ is a voluntary Vice-President Americas and Global of FIN. TL is the Vice-Chairperson of Hong Kong MPS & Rare Genetic Diseases Mutual Aid Group. MP is the voluntary President of FIN and the Greek Lysosomal Organization; FIN received grants from Amicus Therapeutics, Chiesi Pharmaceuticals, Sangamo Therapeutics, Sanofi, Spark Therapeutics and Takeda; FIN received payment for expert testimony from Sanofi and Takeda. TS is a board member of IPA and Chairman of Pompe Deutschland. IPA received grants and Pompe Deutschland received charitable donations from Sanofi. TS received meeting attendance support from Sanofi. IŽ is a board member of IGA and Vice-Chair of the Slovenian Gaucher Association; received meeting and advisory board attendance support from Sanofi. LDB declares no conflict of interest.