Conflict of Interest

VC reports consulting fees and research funding from Sanofi-Genzyme, Takeda, and Amicus. PGP reports consulting fees and research funding to his institution from Sanofi-Genzyme and Takeda. Other authors have no conflict of interest relevant to this work to declare.

Ethical Approval

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.

Informed Consent

Informed consent was obtained from all individual participants included in the study.