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The main causes of acquired anemia can be divided into loss of blood (hemolysis or hemorrhage) or inadequate bone-marrow function (e.g. ineffective erythropoiesis, sideroblastic changes ...
Patients in the EU with Fanconi anaemia (FA) could soon have the first gene therapy option for the disorder, as the EMA starts a review of Rocket Pharma’s RP-L102 candidate. FA is an inherited ...
as well as in anaemia failing an erythropoiesis stimulating agent that requires two or more RBC units over eight week in adult patients with IPSS-R myelodyplastic syndrome with ring sideroblasts ...
Nat Clin Pract Neurol. 2006;2(9):482-493.