Funding for this research was provided by:
Shire International GmbH
Shire Human Genetic Therapies Inc.
Received: 13 June 2018
Accepted: 8 July 2019
First Online: 23 July 2019
Ethics approval and consent to participate
: Informed consent was obtained from all individual participants included in the study. All procedures performed 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. All study materials were approved by appropriate ethics committees (Chesapeake Institutional Review Board for MLD interviews, Ethical & Independent Review Services for MPS II and RTI International for MPS IIIA).
: Not applicable.
: Magdalena Harrington and Jaromir Mikl were employees of Shire (a member of the Takeda group of companies) while conducting this study and during initial manuscript development; Jaromir Mikl was also a shareholder of Shire (a Takeda company). Magdalena Harrington is currently employed by Pfizer and Jaromir Mikl is currently employed by Purdue Pharma. Asha Hareendran and Anne Skalicky are current employees of Evidera; Hilary Wilson was also employed by Evidera while conducting this study and during initial manuscript development. Evidera received a consulting contract from Shire (a Takeda company) to conduct part of the research described in this manuscript; these authors did not receive fees directly from Shire (a Takeda company). Hilary Wilson is now employed by Boehringer Ingelheim Pharmaceuticals, Inc. Marci Clark is employed by RTI Health Solutions, a non-profit organization that provides research consulting services to pharmaceutical and biotechnology companies.