Verma, I. C.
El-Beshlawy, A.
Tylki-Szymańska, A.
Martins, A.
Duan, Y.-L.
Collin-Histed, T.
van der Linde, M. Schoneveld
Mansour, R.
Dũng, V. C.
Mistry, Pramod K. http://orcid.org/0000-0003-3447-6421
Funding for this research was provided by:
Sanofi Genzyme
Article History
Received: 8 November 2021
Accepted: 30 January 2022
First Online: 4 April 2022
Declarations
:
: All patients provided informed consent to participate in the program.
: Consent was obtained to publish images of individuals.
: AM has received consulting fees from Alexion, BioMarin, Chiesi, Sanofi Genzyme, Takeda, and Ultragenix; travel and congress sponsorship and fees for lectures from Alexion, Biomarin, Sanofi Genzyme, Takeda, and Ultragenix; and research grants from Alexion, BioMarin, Sanofi Genzyme, and Takeda. TCH has received research grants from Sanofi Genzyme and served on advisory boards for Sanofi Genzyme. MSvdL has received consulting fees from Amicus Therapeutics, Gilead Sciences, International Pompe Association, and Sanofi Genzyme. Her work in the Humanitarian Program never has been paid and was done on a voluntary basis. PKM has received research grant and travel support from Sanofi Genzyme. All other authors declare no competing interests.