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10.1016/j.bioorg.2023.106550 70 LiuY.ZhaoH.LiL.YangB.YueY.LiM.et al (2023b)

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Examples included: Paroxysmal Nocturnal Haemoglobinuria (PNH) Immune Thrombocytopenia (ITP) Hemophagocytic Lymphohistiocytosis (HLH) Myelofibrosis Haemophilia von Willebrand disease, and other inherited bleeding disorders These studies examined treatment patterns, clinical outcomes, disease burden, healthcare utilization, and patient experiences outside controlled clinical trial environments

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