Sebelipase alfa enzyme replacement therapy in Wolman disease: a nationwide cohort with up to ten years of follow-up.
Tanguy DemaretFlorence LacailleCamille WickerJean-Baptiste ArnouxJuliette BouchereauClaire BellocheCyril GitiauxDavid GreventChristine BroissandDalila AdjaoudMarie-Thérèse Abi WardeDominique PlantazSoumeya BekriPascale de LonlayAnaïs BrassierPublished in: Orphanet journal of rare diseases (2021)
Early ERT initiation allowed 100% survival with positive outcomes. Very long-term follow-up and hematopoietic stem cell transplantation while on ERT should be evaluated to strengthen the benefits of sebelipase alfa.