Document is current
Any future updates will be listed below
-
Authors
Zimran, Ari
Revel-Vilk, Shoshana
Dinur, Tama
Istaiti, Majdolen
Botha, Jaco
Lukina, Elena
Giraldo, Pilar
Deegan, Patrick
Dahl, Stephan vom
-
Funding
Funding for this research was provided by:
The Gaucher Outcome Survey is sponsored by Takeda Pharmaceutical Company Limited (The Gaucher Outcome Survey is sponsored by Takeda Pharmaceutical Company Limited)
- License Information
-
More Information
Article History
Received: 19 February 2024
Accepted: 11 November 2024
First Online: 29 January 2025
Declarations
:
: The GOS was approved by the ethics institutional review boards of the participating centers. Furthermore, this registry was compliant with relevant global and local regulations and best practices: Good Pharmacoepidemiological Practice and Good Research for Comparative Effectiveness principles. The relevant principles of the International Council for Harmonisation Good Clinical Practice guidelines (ICH E6) were followed as appropriate for an observational registry, consistent with the Declaration of Helsinki. All participants gave written informed consent. ClinicalTrials.gov, NCT03291223.
: Not applicable.
: AZ has received honoraria from BioEvents, Pfizer, and Takeda, and has received consultancy fees from Insightec, NLC Pharma, Prevail Therapeutics, and Takeda. SR-V has received research grants, speaker fees, and travel support from Pfizer, Sanofi Genzyme, and Takeda. JB is an employee of Takeda Pharmaceuticals International AG, Zurich, Switzerland and a stockholder of Takeda Pharmaceuticals Company, Limited. PG has received financial compensation for presentations, advisory boards, and research projects from Pfizer, Sanofi Genzyme, and Takeda. The financial contributions are destined to research in Gaucher disease and other lysosomal diseases through the FEETEG. PD has received speaker honoraria and institutional research support from Sanofi and Takeda. SvD has received compensation for presentations, advisory boards, and research projects from Pfizer, Sanofi Genzyme, and Takeda Shire. TD and MI declare that they have no competing interests.
