Article History
Received: 6 December 2018
Accepted: 12 December 2018
First Online: 7 January 2019
Ethics approval and consent to participate
: Legal authorizations were obtained from the Comité consultatif sur le traitement de l’information en matière de recherche dans le domaine de la santé (n°14.324bis) and the Comité de Protection des personnes Paris VI, according to the relevant French legislation.All patients signed written consent after specific oral and written information, for this research and its publication.
: All patients signed written consent after specific oral and written information, for this research and its publication.
: Inserm U974 research team received financial support from Shire, AFM-Téléthon, SNFMI (Société Nationale Française de Médecine Interne) and VML (Vaincre les Maladies Lysosomales) for this study.Wladimir Mauhin: Travel fees and accommodations: Shire, Orphan Europe, Amicus, Sanofi-Genzyme, honorarium: Shire.Olivier Lidove: Travel fees, accommodations and honorarium: Shire, Sanofi-Genzyme; honorarium: Amicus.Olivier Benveniste: Travel fees Shire, LFB, CSL Behring; honorarium: Novartis, Neovacs, LFB.
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