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Article History

Received: 7 November 2018

Accepted: 20 February 2019

First Online: 14 March 2019

Ethics approval and consent to participate

: Ethics approval is not mandatory for non-interventional studies conducted in France and Spain. Data privacy collection approval was obtained. An information sheet was provided to patients and verbal consent was obtained in compliance with French and Spanish law for such a study.

: Not applicable.

: All authors except GT, BF and MS received payment from Orphan Europe directly or indirectly through their institution/association for participation in the registry. VV is currently an employee of Sanofi Genzyme. He performed most of this work while he was at Necker-Enfants Malades University Hospital. LD has a conflict of interest with Orphan Europe. FM has received an honorarium from Orphan Europe. The other authors have no conflicts of interest to declare.

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