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Research Articles

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Compiled for your convenience are articles on bone marrow failure research and treatment from the world’s major journals on hematology / oncology.

Article Title Original Publication Date Journal Bone Marrow Disease
To Target the Untargetable: Elucidation of Synergy of APR-246 and Azacitidine in TP53 Mutant Myelodysplastic Syndromes and Acute Myeloid Leukemia Jun 2020 Haematologica Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS)
Myelodysplastic Syndromes: Moving Towards Personalized Management May 2020 Haematologica Myelodysplastic Syndromes (MDS)
Detectable mutations precede late myeloid neoplasia in aplastic anemia Feb 2021 Haematologica Aplastic Anemia
Significant hemolysis is not required for thrombosis in paroxysmal nocturnal hemoglobinuria. Mar 2019 Haematologica Paroxysmal Nocturnal Hemoglobinuria (PNH)
Conditional survival and standardized mortality ratios of patients with severe aplastic anemia surviving at least one year after hematopoietic cell transplantation or immunosuppressive therapy Jun 2023 Haematologica Aplastic Anemia
Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria Aug 2023 Haematologica Paroxysmal Nocturnal Hemoglobinuria (PNH)
Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria Oct 2020 Haematologica Paroxysmal Nocturnal Hemoglobinuria (PNH)
Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study Apr 2019 Haematologica Aplastic Anemia
Characterization of breakthrough hemolysis events observed in the phase 3 randomized studies of ravulizumab versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria Jan 2020 Haematologica Paroxysmal Nocturnal Hemoglobinuria (PNH)
Time without transfusion reliance: a novel patient-centric metric for new therapies in myelodysplastic syndromes Apr 2023 Haematologica Myelodysplastic Syndromes (MDS)