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The LSD1 inhibitor RN-1 recapitulates the fetal pattern of hemoglobin synthesis in baboons (P. anubis).
The syndrome of hemophagocytic lymphohistiocytosis in primary immunodeficiencies: implications for differential diagnosis and pathogenesis.
Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia.
Response to "efficacy and safety of sildenafil for the treatment of severe pulmonary hypertension in patients with hemoglobinopathies: results from a long-term follow up " Haematologica 2014;99(2):e17-18.
Non-transfusion-dependent thalassemias.
A randomized, placebo-controlled trial of arginine therapy for the treatment of children with sickle cell disease hospitalized with vaso-occlusive pain episodes.
Sildenafil therapy in thalassemia patients with Doppler-defined risk of pulmonary hypertension.
Relationship between labile plasma iron, liver iron concentration and cardiac response in a deferasirox monotherapy trial.
A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload.
Italian Society of Hematology guidelines for thalassemia and non-invasive iron measurements.