Funding for this research was provided by:
BioMarin Pharmaceutical (Not applicable)
Received: 24 January 2019
Accepted: 26 June 2019
First Online: 8 July 2019
Ethics approval and consent to participate
: The secondary research is based on survey data and thus no ethics approval was required. The secondary research was based on survey data in which the participants agreed to answer. No formal consent was required.
: Not applicable.
: ES reports consulting fees from Shapiro Neuropsychology Consulting, LLC. CML has received honoraria for speakers’ fees from Actelion, Genzyme, and Shire HGT; all fees are donated to the CML Medical Foundation for Research and Genetic Diagnosis Support for families with unknown genetic disorders. NM is a consultant for BioMarin, Shire, Genzyme Sanofi, Lysogene, and SOBI; has received grants/research support from BioMarin, Shire, and Genzyme Sanofi; and has received honoraria and travel grants from BioMarin, Shire, Genzyme Sanofi, Actelion, and Amicus. CO’N and NOM declare they have no competing interests. SV has received research support from Alexion Pharmaceuticals; has received travel grants from Actelion, BioMarin, Genzyme, and Shire; and has served as an advisory board member for Vtesse.