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Article History

Received: 19 August 2019

Accepted: 24 March 2020

First Online: 19 April 2020

Ethics approval and consent to participate

: Not applicable.

: Not applicable.

: The content of this manuscript is based on discussions during a meeting that was coordinated and funded by BioMarin Pharmaceutical Inc. BioMarin also provided a review of the manuscript. RG, PH, S-PL and MS received funding from BioMarin Pharmaceutical Inc. to participate in this meeting. In addition, RG received speaker honoraria, advisory board and consultancy fees, investigator fees, research grants or travel grants to participate in scientific meetings from Amicus, Actelion, BioMarin, GC Pharma, Inventiva, JCR, Lysogene, PTC, RegenxBio, Sangamo, Sanofi, Sobi, Takeda, and Ultragenyx. MS reports board membership and grants and payments (e.g. for lectures, speakerships, honoraria, travel/accommodation) from Shire, Genzyme, Chiesi, Ultragenix, and BioMarin. PH has been a consultant for BioMarin, Shire, Genzyme, Chiesi, Inventiva, Paradigm, Ultragenyx, SOBI, JCR, Denali, Orphazyme, RegenXbio, and Sangamo, he received grants from BioMarin, payments (lectures, speakerships, honoraria) from BioMarin, Chiesi, Ultragenyx, and Orphazyme and travel, accommodations, and/or payments for meeting expenses from BioMarin, Shire, Genzyme, Chiesi, Inventiva, Ultragenyx, SOBI, and RegenXbio.