Early access program for danicopan (ALXN2040) as add-on treatment to eculizumab or ravulizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH): Preliminary data from the Italian real-world survey.
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DOI: 10.1182/blood-2025-4665
Abstract: Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematopoietic disease characterized by complement-mediated intravascular hemolysis. Although C5 inhibitors (C5i) secure effective control of intravascular hemolysis (IVH), a variable degree of consequent extravascular hemolysis (EVH) might… read more here.
Keywords: range; pnh; hgb; danicopan ... See more keywords