Häberle, Johannes
Chakrapani, Anupam
Ah Mew, Nicholas
Longo, Nicola http://orcid.org/0000-0002-3677-1216
Funding for this research was provided by:
Orphan Europe
Article History
Received: 29 June 2018
Accepted: 26 November 2018
First Online: 6 December 2018
Ethics approval and consent to participate
: Not applicable.
: Not applicable.
: JH has received travel support and/or honoraria from Hyperion Therapeutics/Horizon Pharma, Lucane Pharma, Nutricia Metabolics, Orphan Europe Recordati Group, and Swedish Orphan Biovitrum (Sobi). NL has received travel support from Orphan Europe, receives support for clinical trials from Horizon Pharma, and is a consultant for Moderna Therapeutics and HemoShear Therapeutics. AC has received honoraria, lecture fees and travel support from Orphan Europe. NAM has received travel support from Orphan Europe and Horizon Pharma, and a grant from Recordati Rare Diseases Group USA.
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