Wenninger, Stephan http://orcid.org/0000-0001-8407-3642
Gutschmidt, Kristina http://orcid.org/0000-0001-9891-5919
Wirner, Corinna
Einvag, Krisztina http://orcid.org/0000-0002-7291-5408
Montagnese, Federica
Schoser, Benedikt http://orcid.org/0000-0002-2757-8131
Funding for this research was provided by:
Ludwig-Maximilians-Universität München
Article History
Received: 22 November 2020
Revised: 12 February 2021
Accepted: 14 February 2021
First Online: 24 February 2021
Compliance with ethical standards
:
: All authors report no disclosures regarding this study. SW has received research grant by the DGM—Deutsche Gesellschaft für Muskelkranke e.V. He has served on advisory boards for Alexion Pharma, CSL Behring and Sanofi Genzyme GmbH. He received funding for travel or speaker Honoraria from Sanofi-Aventis Germany GmbH; SH Glykogenose Gesellschaft; AbbVie Germany GmbH; Recordati Pharma GmbH; CSLBehring GmbH; Alexion Pharma GmbH; Desitin Germany; Akcea GmbH. BS has served on advisory boards for Amicus Therapeutics, Audentes, Nexien, Spark, and Sanofi Genzyme; he has undertaken contracted Page 21/25 unrestricted research for Greenovation Biopharm, Sanofi Genzyme, Amicus; and has received honoraria from Kedrion and Alexion, and travel expenses from Amicus and Sanofi Genzyme. FM received funding for travel or speaker Honoraria from Sanofi-Aventis Germany GmbH and SH Glykogenose Gesellschaft. The other authors have no conflicts of interest to declare.
: The assessments in this study were performed within the national POMPE Registry program, approved by the Medical Ethics Committee of the Landesaerztekammer Rheinland-Pfalz (no. 7/04929) and conducted in accordance with the ethical standards recognized internationally.