[Rinsho ketsueki] The Japanese journal of clinical hematologyHumansHemoglobinuriaParoxysmalComplement Inactivating AgentsComplement C5
[Challenges in optimizing targeted complement inhibitor therapy for paroxysmal nocturnal hemoglobinuria in an expanding treatment landscape].
Shikiko Ueno
Published: 202510.11406/rinketsu.66.915
Abstract
The advent of the C5 inhibitor eculizumab marked a paradigm shift in the management of PNH, transforming it from "a rare disease with a poor prognosis" to "a manageable disease". However, emerging challenges such as primary nonresponse due to C5 gene…
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