Paroxysmal Nocturnal Hemoglobinuria (PNH) research hub

A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor

This study looks at a new drug, ALN-CFB, for people with a blood condition called PNH who have ongoing anaemia despite current treatment. It aims to see how safe and effective the new drug is compared to a dummy drug.