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Article History

Received: 19 November 2020

Accepted: 25 February 2021

First Online: 10 March 2021

Declarations

:

: This study has been carried out in accordance with The Code of Ethics of the World Medical Association (Declaration of Helsinki). The study was initiated and carried out by patient organization HCU Network Australia, data were anonymous and no ethics committee approval was sought. The “Kantonale Ethikkommission Zürich” raised no concern regarding statistical analysis of the anonymised survey data.

: Not applicable.

: Not applicable.

: MH, AAMM, MAL, VK, TM and FB declare no conflicts of interest regarding the contents of this manuscript. MH has received research grants from Nutricia Metabolics and SOBI and honoraria for lectures or scientific advice from Nutricia Metabolics, Recordati Rare Disease Foundation, Shire, Aeglea and Sanofi Genzyme. AAMM has received honoraria for lectures from Recordati & Nutricia and Advisory Board fees from Nutricia. FB has received research grants from Nutricia Metabolics. VK declares that Charles University-First Faculty of Medicine received in the past a partial reimbursement for preclinical testing of OT-58 from Orphan Technologies. HCU Network Australia has received sponsorship from Orphan Technologies and support from the Recordati Rare Disease Foundation, Orphan Europe, Recordati Rare Diseases Australia, Nutricia Metabolics, PIAM, BioElectron, Vitaflo, MetaX, Erytech, Recordati Rare Diseases Brasil and CMW SAÚDE for patient expert meetings.