Gittoes, Neil
Rejnmark, Lars
Ing, Steven W.
Brandi, Maria Luisa
Björnsdottir, Sigridur
Hahner, Stefanie
Hofbauer, Lorenz C.
Houillier, Pascal
Khan, Aliya A.
Levine, Michael A.
Mannstadt, Michael
Shoback, Dolores M.
Vokes, Tamara J.
Zhang, Pinggao
Marelli, Claudio
Germak, John
Clarke, Bart L.
Article History
Received: 19 November 2020
Accepted: 26 October 2021
First Online: 20 November 2021
Declarations
:
: The study protocol was reviewed and approved by the Institutional Review Board/Independent Ethics Committee/Research Ethics Board at each participating site. Central Institutional Review Boards were utilized by 1 UK, 4 Swedish, and 19 US sites; Institutional Review Boards were utilized by 27 US sites. The patient, the parent(s) of a pediatric patient, or a legally authorized representative is required to provide written informed consent. The study is being conducted in accordance with Good Pharmacoepidemiology Practice as described by the International Society for Pharmacoepidemiology.
: Not applicable
: NG and SH have served as an advisory board member and consultant for Shire, a Takeda company. LR has served as an advisory board member, research investigator, and speaker for Shire, a Takeda company. SWI has served as an advisory board member and research investigator for Shire, a Takeda company, and served as research investigator for Amgen Inc., Chugai Pharmaceutical Co., Ltd, Radius Health Inc., and Ultragenyx Pharmaceutical. SB and AAK have served as research investigators for Shire, a Takeda company. MLB has served as a research investigator and speaker for Shire, a Takeda company. LCH has served as a research investigator and received honoraria for advisory boards from Shire, a Takeda company, and received honoraria for advisory boards from Ascendis Pharma, Alexion Pharmaceuticals Inc., Amgen Inc., Kyowa Kirin International, and USB. PH has served as an advisory board member, consultant, research investigator, and speaker for Shire, a Takeda company. MAL, DMS, and TV have served as an advisory board member, consultant, and research investigator for Shire, a Takeda company. PZ and JG are employed by Shire Human Genetic Therapies, Inc., a Takeda company, Lexington, MA, USA. MM has served as an advisory board member, consultant, and research investigator for Shire, a Takeda company, as consultant and research investigator for Chugai Pharmaceutical Co., Ltd, and as consultant for Amolyt Pharma. CM is employed by Takeda Pharmaceuticals International AG, Zurich, Switzerland. BLC has served as consultant and research investigator for Shire, a Takeda company, and has received grant funding from Ascendis Pharma and Chugai Pharmaceutical Co., Ltd.