Hennermann, Julia B.
Guffon, Nathalie
Cattaneo, Federica
Ceravolo, Ferdinando
Borgwardt, Line
Lund, Allan M.
Gil-Campos, Mercedes
Tylki-Szymanska, Anna
Muschol, Nicole M.
Article History
Received: 17 December 2019
Accepted: 14 September 2020
First Online: 29 September 2020
Ethics approval and consent to participate
: Before the study commences, the protocol will be submitted for ethics approval according to the requirements of each country. Informed consent will be obtained from each patient prior to the collection of any data.
: Not applicable.
: JBH received consulting fees and travel expenses from Chiesi Farmaceutici S.p.A., honoraria and travel expenses from Amicus and Shire. NG is a member of an advisory board and received consulting fees from BioMarin, Chiesi Farmaceutici S.p.A, Sanofi Genzyme, Shire, SOBI, and Ultragenyx Pharmaceutical Inc. FeCa and FeCe are employed at Chiesi Farmaceutici S.p.A. LB received consulting fees from Chiesi Farmaceutici S.p.A. AML received consulting fees from Alexion, BioMarin, Chiesi Farmaceutici S.p.A., Sanofi Genzyme, and Shire. MG-C and AT-S were members of an advisory board for Chiesi Farmaceutici S.p.A. NMM is a consultant for BioMarin, Sanofi Genzyme, Lysogene, Shire, and SOBI, has received grants/research support from Amicus, BioMarin, Sanofi Genzyme, and Shire, and has received honoraria and/or travel grants from Actelion, Amicus, BioMarin, Chiesi Farmaceutici S.p.A., Sanofi Genzyme, and Shire.