Congenital amegakaryocytic thrombocytopenia 1

A rare inherited bone marrow failure syndrome, in which the cause of the disease is a variation in the MPL gene. It is characterized by an isolated and severe decrease in the number of platelets and megakaryocytes during the first years of life that develops into bone marrow failure with pancytopenia later in childhood.

Also known as: CAMT1, amegakaryocytic thrombocytopenia, congenital 1, thrombocytopenia, congenital amegakaryocytic, thrombocytopenia congenital amegakaryocytic

5 clinical trials for this condition and its sub-types, 3 tagged with Congenital amegakaryocytic thrombocytopenia 1 itself.