Real-World check: can new drug free PNH patients from transfusions?

NCT ID NCT07229235

Summary

This study aims to see how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) in everyday Italian clinics. It will follow about 50 adult patients for up to two years to see if the treatment helps raise their hemoglobin levels and allows them to avoid blood transfusions. The study will also track side effects and how patients feel, using data from their regular medical visits.

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