Giugliani, Roberto http://orcid.org/0000-0001-9655-3686
Gonzalez-Meneses, Antonio
Scarpa, Maurizio
Burton, Barbara
Wang, Raymond
Martins, Esmeralda
Oussoren, Esmeralda
Hennermann, Julia B.
Chabrol, Brigitte
Grant, Christina L.
Sun, Angela
Durand, Consuelo
Hetzer, Joel
Malkus, Betsy
Marsden, Deborah
Merritt II, J. Lawrence
Funding for this research was provided by:
Ultragenyx Pharmaceutical (Ultragenyx Pharmaceutical)
Article History
Received: 17 December 2023
Accepted: 30 March 2024
First Online: 7 May 2024
Declarations
:
: The protocol was reviewed and approved by the institutional review boards at respective sites. Prior to any research-related procedures, participants provided written informed consent or, in the case of participants < 18 years of age (or 16 years, depending on the region) or participants > 18 years of age with cognitive deficiencies, provided written assent (if required), with written informed consent provided by a legally authorized representative after the nature of the DMP had been explained.
: All authors approved the manuscript for publication.
: Joel Hetzer, Betsy Malkus, Deborah Marsden, and J. Lawrence Merritt II are employees and shareholders of Ultragenyx Pharmaceutical Inc.