Kishnani, Priya S.
Shohet, Simon http://orcid.org/0009-0003-0901-5233
Raza, Syed
Hummel, Noemi
Castelli, Jeffrey P.
Sitaraman Das, Sheela
Jiang, Heng
Kopiec, Agnieszka
Keyzor, Ian
Hahn, Andreas
Funding for this research was provided by:
Amicus Therapeutics (Amicus Therapeutics)
Article History
Received: 21 August 2023
Accepted: 10 January 2024
First Online: 31 January 2024
Declarations
:
: This study used data from the PROPEL phase 3 study. PROPEL was designed and monitored in accordance with the ethical principles of Good Clinical Practice guidelines and the Declaration of Helsinki. The clinical study protocol was reviewed and approved by the appropriate independent ethics committee or institutional review board at each study site. All participants provided written informed consent before participating in the study.
: Not applicable.
: SiS, IK, JC, and ShSD are employees of and hold stock in Amicus Therapeutics. SR is a former employee of Amicus Therapeutics. NH, AK, and HJ are employees of Certara; Certara is a paid consultant to Amicus Therapeutics. PK has received research/grant support from Amicus Therapeutics and Sanofi Genzyme; has received consulting fees and honoraria from Amicus Therapeutics, Sanofi Genzyme, Maze Therapeutics, Bayer and Asklepios Biopharmaceutical, Inc. (AskBio); is a member of the Pompe and Gaucher Disease Registry Advisory Board for Sanofi Genzyme, Pompe Disease Advisory Board for Amicus Therapeutics, and Advisory Board for Baebies; has equity with Maze Therapeutics and has held equity in Asklepios Biopharmaceuticals, and may receive milestone payments related to that equity in the future. AH received honorariums for consulting activities from Sanofi-Aventis, Amicus, and Avrobio, and grants for research projects from Sanofi-Aventis.