Thrombopoietin mutation in congenital amegakaryocytic thrombocytopenia treatable with romiplostim

Pecci, A; Ragab, I; Bozzi, V; De Rocco, D; Barozzi, S; Giangregorio, T; Ali, H; Melazzini, F; Sallam, M; Alfano, C; Pastore, A; Balduini, CL; Savoia, A

Savoia, A (reprint author), Inst Maternal & Child Hlth IRCCS Burlo Garofolo, Trieste, Italy.; Savoia, A (reprint author), Univ Trieste, Dept Med Sci, Trieste, Italy.

EMBO MOLECULAR MEDICINE, 2018; 10 (1): 63

Abstract

Congenital amegakaryocytic thrombocytopenia (CAMT) is an inherited disorder characterized at birth by thrombocytopenia with reduced megakaryocytes, wh......

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