Sebelipase alfa enzyme replacement therapy in Wolman disease: a nationwide cohort with up to ten years of follow-up
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P. de Lonlay | A. Brassier | J. Arnoux | S. Bekri | D. Plantaz | J. Bouchereau | C. Gitiaux | F. Lacaille | D. Grevent | C. Broissand | C. Wicker | Tanguy Demaret | D. Adjaoud | Marie-Thérèse Abi Wardé | C. Belloche | T. Demaret