Dahir, Kathryn McCrystal https://orcid.org/0000-0002-9980-9138
Dunbar, Nancy S. https://orcid.org/0000-0002-1783-4605
Article History
Accepted: 17 February 2025
First Online: 18 March 2025
Declarations
: KD is a clinical trial investigator for Alexion, Regeneron, Ultragenyx, Kyowa Kirin, Sanofi. ND serves as a clinical trial investigator for Alexion and Ultragenyx, and as consultant and speaker board member for Alexion, Kyowa Kirin, and Biomarin.
: Both authors are clinical trial investigators for Alexion so this must be disclosed. See disclosures in the manuscript. However this review article was not funded by Alexion or any other organization.