Moisan, Lina
Iannuzzi, David
Maranda, Bruno
Campeau, Philippe M.
Mitchell, John J. http://orcid.org/0000-0002-6055-6858
Funding for this research was provided by:
BioMarin Pharmaceutical
Article History
Received: 23 April 2020
Accepted: 14 September 2020
First Online: 29 September 2020
Ethics approval and consent to participate
: The study protocol was approved by the institutional review board at McGill University. All patients (or their guardians) provided written informed consent before study entry. The study was conducted in accordance with the Declaration of Helsinki and International Conference of Harmonization Good Clinical Practice guidelines.
: Not applicable.
: JJM has been an investigator on clinical trials with BioMarin, Takeda, Ultragenyx, Enzyvant, and Sanofi-Genzyme, and has received consulting fees from BioMarin, Ultragenyx, Enzyvant and Sanofi-Genzyme; PMC is an investigator on the BioMarin MARS study and has received consulting fees from BioMarin; BM is an investigator on the BioMarin MARS study; all other authors have no conflicts of interest to declare.