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Article History

Received: 20 February 2018

Accepted: 18 July 2018

First Online: 31 July 2018

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.Foudil Lamari: Travel fees and accommodations: Shire, Sanofi-Genzyme; Actelion Pharmaceuticals; honorarium Actelion Pharmaceuticals.Catherine Caillaud: Travel fees and accommodations: Sanofi-Genzyme; honorarium: Shire.Claire Douillard: Travel fees and accommodations: Shire, Sanofi-Genzyme.Bertrand Dussol: Honorarium: Amicus (member of the scientific board), Novartis (lectures), Travel fees: Genzyme-Sanofi, honorarium: Novartis.Vanessa Leguy-Seguin: Travel fees and accommodations: Shire, Sanofi-Genzyme.Esther Noel: Travel fees Shire, Sanofi-Genzyme; honorarium: Amicus.Thierry Zenone: Travel fees and accommodations: Sanofi-Genzyme; travel fees and honorarium: Shire.François Maillot: Travel fees Shire, Sanofi-Genzyme; honorarium: Sanofi-Genzyme.Gérard Besson: honorarium: Shire.Fabien Labombarda: Travel fees and accommodations: Shire, Genzyme-Sanofi.Agathe Masseau: Travel fees and accommodations: Shire, Sanofi-Genzyme.Christian Lavigne: Travel fees and accommodations: Shire, Sanofi-Genzyme.Didier Lacombe: honorarium Genzyme Sanofi, Shire, Biomarin.Eric Hachulla: Honorarium: Sanofi-Genzyme, Shire; research grants: Sanofi-Genzyme; travel fees and accommodation: Sanofi-Genzyme, Shire.Olivier Benveniste: Travel fees Shire, LFB, CSL Behring; honorarium: Novartis, Neovacs, LFB.Damien Amelin, Louiza Arouche-Delaperche, Gaelle Dzangue-Tchoupou, Kim Heang Ly, Marjolaine Willems, Roseline Froissart, Marie Bénédicte Matignon, Jean-Marc Ziza, Federico Mingozzi and Pauline d’Halluin declare no conflicts of interest.

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