Pabinger, Ingrid
Ayash-Rashkovsky, Mila
Escobar, Miguel http://orcid.org/0000-0002-2944-0240
Konkle, Barbara A. http://orcid.org/0000-0002-3959-8797
Mingot-Castellano, MarĂa Eva http://orcid.org/0000-0001-9083-855X
Mullins, Eric S.
Negrier, Claude http://orcid.org/0000-0002-2905-055X
Pan, Luying
Rajavel, Kavitha
Yan, Brian
Chapin, John http://orcid.org/0000-0003-3365-7757
Funding for this research was provided by:
Takeda Development Center Americas, Inc. Cambridge, MA, USA
Article History
Received: 13 June 2023
Revised: 8 January 2024
Accepted: 12 January 2024
First Online: 14 February 2024
Competing interests
: MA-R was an employee of Takeda Development Center Americas, Inc. and Takeda stockholder at the time the study was conducted. JC, LP, KR and BY employees of Takeda Development Center Americas, Inc., and Takeda stockholders. ME honoraria for advisory boards or consulting (NovoNordisk, Takeda, Bayer, CSL Behring, Genentech/Roche, UniQure, Biomarin, Sanofi, Pfizer, Hemobiologics/LFB, NHF); research support (NovoNordisk, Uniqure, Baxalta, Bayer, Sanofi, Takeda, Pfizer, and OPKO Biologics). BK research support (CSL Behring, Pfizer, Sanofi, Spark, Takeda, and Uniqure); consulting fees (BioMarin, Novo Nordisk, Pfizer, Regeneron, Sanofi, Takeda, and Uniqure). MEM-C: none. ESM: research support (Takeda); consulting fees (Takeda). CN: research support (Roche, Sobi, and Takeda); advisory board/consulting fees (Bayer, Biomarin, CSL Behring, Novo Nordisk, Pfizer, Roche, Sanofi, Sobi, Takeda, and UniQure). IP research support (CSL Behring and Sobi and Roche Diagnostics to the Medical University of Vienna); honoraria for lectures, advisory boards, or participating as an advisor (CSL Behring, Takeda, Roche, Sobi, NovoNordisk, Bayer, Pfizer, Sandoz).
: This study was conducted in accordance with the International Council for Harmonization Guideline for Good Clinical Practice, the Declaration of Helsinki, and applicable international, national, and local regulatory requirements. All participants provided written informed consent.