Funding for this research was provided by:
Received: 9 April 2019
Accepted: 11 June 2019
First Online: 2 July 2019
Ethics approval and consent to participate
: Informed consent was obtained either from the patient or their parents/legal guardians according to local laws. The participating site or physician was responsible for obtaining ethical approval.
: Not applicable.
: <b>MP, KJ, PK</b> and <b>MK</b> have received honoraria for talks and travel reimbursement from Actelion Pharmaceuticals Ltd.<b>VM</b> has received consultancy fees, honoraria and travel reimbursement from Actelion Pharmaceuticals Ltd., Alexion Pharma GmbH, Sanofi-Genzyme and Shire.<b>JT</b> is an employee of Syntax for Science S.L.<b>SAK</b> is an employee of Actelion Pharmaceuticals Ltd., a Janssen Pharmaceutical Company of Johnson & Johnson.