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Authors
Lanar, Sally http://orcid.org/0000-0001-6914-9004
Parker, Samantha
O’Neill, Cara
Marrel, Alexia
Arnould, Benoit
Héron, Bénédicte
Muschol, Nicole
Wijburg, Frits A.
Chakrapani, Anupam
Olivier, Sophie
Aiach, Karen
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Funding
Funding for this research was provided by:
Lysogene
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Article History
Received: 13 September 2021
Accepted: 6 February 2022
First Online: 22 February 2022
Declarations
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: The clinical trial protocol, including description of the qualitative interviews, was approved by all relevant independent ethics committees and institutional review boards at each site: Academic Medical Center (Netherlands), CHU Paris Est—Hopital d'Enfants Armand-Trousseau (France), Great Ormond Street Hospital (United Kingdom), and University Medical Center Hamburg—Eppendorf (Germany).
: Not applicable.
: SL, AM and BA are employees of ICON plc and paid consultants for Lysogene. SP and SO were employees of Lysogene at the time of the study. KA is the founder, chair and CEO of Lysogene. NH has received grant/research support from BioMarin, Sanofi Genzyme and Shire/Takeda, she received consulting fees and/or honoraria/travel support from Actelion, Amicus, BioMarin, Chiesi, JCR Pharmaceuticals, Lysogene, Sanofi Genzyme, Shire/Takeda and Sobi. AC works for Great Ormond Street Hospital which received funding from Lysogene to conduct research. CO is a paid consultant for Lysogene. FW has served as a consultant for Orchard Therapeutics and received travel grants from Lysogene. BH is an expert consultant for Lysogene. She has received honoraria for advisory boards from Orchard Therapeutics, Actelion, and Shire/Takeda, Orphazyme; received honoraria/travel support from Actelion, BioMarin, Shire/Takeda; is principal investigator for Abeona, Orphazyme, Lysogene, Mallincrodt, Idorsia, and Chiesi studies.
