Thrombopoietin mutation in congenital amegakaryocytic thrombocytopenia treatable with romiplostim

Abstract Congenital amegakaryocytic thrombocytopenia (CAMT) is an inherited disorder characterized at birth by thrombocytopenia with reduced megakaryocytes, which evolves into generalized bone marrow aplasia during childhood. Although CAMT is genetically heterogeneous, mutations of MPL, the gene enc...

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Bibliographic Details
Main Authors: Alessandro Pecci, Iman Ragab, Valeria Bozzi, Daniela De Rocco, Serena Barozzi, Tania Giangregorio, Heba Ali, Federica Melazzini, Mohamed Sallam, Caterina Alfano, Annalisa Pastore, Carlo L Balduini, Anna Savoia
Format: Article
Language:English
Published: Wiley 2018-01-01
Series:EMBO Molecular Medicine
Subjects:
MPL
Online Access:https://doi.org/10.15252/emmm.201708168