Sebelipase alfa enzyme replacement therapy in Wolman disease: a nationwide cohort with up to ten years of follow-up
塞贝利酶α酶替代疗法治疗沃尔曼病:一项全国性队列研究,随访时间长达十年
期刊:Orphanet Journal of Rare Diseases
影响因子:3.5
doi:10.1186/s13023-021-02134-3
Demaret, Tanguy; Lacaille, Florence; Wicker, Camille; Arnoux, Jean-Baptiste; Bouchereau, Juliette; Belloche, Claire; Gitiaux, Cyril; Grevent, David; Broissand, Christine; Adjaoud, Dalila; Abi Warde, Marie-Thérèse; Plantaz, Dominique; Bekri, Soumeya; de Lonlay, Pascale; Brassier, Anaïs