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PNH (Paroxysmal Nocturnal Hemoglobinuria) is a rare bone marrow failure disorder disease and is listed in the "First National Rare Disease Catalog" in China. The study clarified the significant clinical and economic burden of the disease and showed that glucocorticoid treatment, including prednisone and dexamethasone while widely used, had limited efficacy, and present safety issues that may lead to incomplete control of symptoms. These results established the need for newer, more innovative treatment strategies to improve the health-related quality of life of PNH patients.
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