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Master Thesis

Le développement de pegcétacoplan dans l'hémoglobinurie paroxystique nocturne

Abstract : Paroxysmal nocturnal hemoglobinuria is a rare disease diagnosed with hemolytic anemia, marrow failure or episodes of venous thrombosis. Other symptoms can also appear. The only curative therapy is a bone marrow transplantation for patients with severe aplastic anemia. Currently two treatments are available, however, unmet clinical needs remain. Pegcetacoplan is a drug in development which may solve some of these unmet needs and also improve patients’ quality of life. This thesis aims to describe the pharmaceutical development and market access of pegcetacoplan in paroxysmal nocturnal hemoglobinuria.
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Master Thesis
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Submitted on : Friday, February 4, 2022 - 12:38:53 PM
Last modification on : Tuesday, February 8, 2022 - 3:38:35 AM
Long-term archiving on: : Thursday, May 5, 2022 - 6:59:41 PM


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  • HAL Id : dumas-03557474, version 1



Oriane Courbier. Le développement de pegcétacoplan dans l'hémoglobinurie paroxystique nocturne. Sciences du Vivant [q-bio]. 2021. ⟨dumas-03557474⟩



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