Received: 23 October 2017
Accepted: 12 January 2018
First Online: 1 February 2018
Ethics approval and consent to participate
: Not applicable
: Patients n°1 and n°2 - followed by Dr. Nadjar- have both given their oral consent to have their personal data published in an anonymized fashion.
: Ana Gales, Marion Masingue, Stephanie Millecamps, Stephane Giraudier, LaureGrosliere, Claude Adam and Vincent Navarro, report no disclosures.Claudio Salim, one of the co-authors of this review is employed by Orphan Europe, the marketing authorization holder of Cystadane (ie, anhydrous betaine).Yann Nadjar received speech honoraria from Actelion and Orphan Europe; and received travel funding from Actelion, Shire and Genzyme.
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