Funding for this research was provided by:
Received: 17 July 2020
Accepted: 13 October 2020
First Online: 6 January 2021
Change Date: 2 March 2021
Change Type: Correction
Change Details: A Correction to this paper has been published:
Change Details: https://doi.org/10.1186/S13023-021-01753-0
Ethics approval and consent to participate
: Both studies were conducted in accordance with Good Clinical Practice, the Declaration of Helsinki, and the moral, ethical, and scientific principles that justify medical research. The protocols, amendments, and informed consent forms were approved by an institutional review board or independent ethics committee prior to initiation of the study at each study site. The patients’ parent/legal guardian(s) provided written informed consent.
: Not applicable.
: S. Vijay, A. Ghosh, and S. Fecarotta have received consultancy fees/honoraria from Alexion Pharmaceuticals, Inc. F. Abel and S. Marulkar are employees of and may own stock/stock options in Alexion Pharmaceuticals, Inc. which sponsored the studies. A. Brassier has received fees and travel support for conferences from Alexion Pharmaceuticals, Inc. S. A. Jones has received consultancy fees, grants, and travel support for conferences from Alexion Pharmaceuticals, Inc.