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Iptacopan Meets Primary End Point of APPOINT-PNH Trial in Paroxysmal Nocturnal Hemoglobinuria

Iptacopan elicited improved hemoglobin levels measuring at least 2 g/dL higher vs baseline, leading to transfusion independence after 24 weeks in approximately 92.2% of patients with complement inhibitor–naïve paroxysmal nocturnal hemoglobinuria.

Trial Shows Iptacopan Lowers Need for Blood Transfusion in Almost All Patients With Paroxysmal Nocturnal Hemoglobinuria

Novartis Phase III APPOINT-PNH trial shows investigational

Data at EBMT show primary endpoint met – estimated 92.2% of complement-inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) achieving 2.

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