Group 1 - The complement system is a major component of innate immunity, and its rapid development is transforming clinical practices in various fields such as hematology, neurology, and nephrology [1] - Significant advancements in the understanding and treatment of rare diseases like Paroxysmal Nocturnal Hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS), generalized Myasthenia Gravis (gMG), and Neuromyelitis Optica Spectrum Disorders (NMOSD) have been made, with breakthrough therapies included in medical insurance [1] - Several complement inhibitors have been approved for use in China, with multiple projects in the research phase, indicating a growing application of these treatments in more diseases [1] Group 2 - AstraZeneca's acquisition of Alexion for $39 billion at the end of 2020 marked its entry into the rare disease sector, with its core product, eculizumab, being the first approved C5 complement inhibitor globally [2] - AstraZeneca plans to advance nearly 10 new products or indications in the rare disease field in China over the next five years, focusing on making medications accessible to all patients in need [2] - The company suggests the establishment of a rare disease special fund by the government to alleviate patient financial burdens and proposes leveraging international experiences to broaden funding channels for long-term medication support [2]

杨春生教授表示，长期以来，gMG患者经胆碱酯酶抑制剂、糖皮质激素、免疫抑制剂、静脉注射免疫 球蛋白、血浆置换以及胸腺切除等可改善病情，但"双达标"的比例有待进一步提高。约20%-30%的患者 经过多重的免疫抑制剂或者激素治疗，仍然会出现症状明显加重或者波动，需要丙球或者血浆置换治 疗，或者因长期应用药物带来很多副作用不能耐受，严重影响患者的生活质量。 "近年来，随着包括补体抑制剂在内的单抗类药物国内上市，全身型重症肌无力（gMG）的治疗正在迈 入疾病症状控制和治疗副作用最小化的'双达标'时代。"近日，天津医科大学总医院神经内科主任医师 杨春生教授接受新京报记者采访时提到了上述现状。他表示，越来越多的新补体药物正在研发和上市过 程中。 全身型重症肌无力是一种神经系统的复杂的、慢性自身免疫疾病。杨春生教授介绍，重症肌无力是神经 系统的罕见病，主要是由B细胞和抗体介导，影响神经肌肉接头的突触后膜，造成神经冲动不能及时有 效传递给肌肉，引起骨骼肌的易疲劳，临床上表现为晨轻暮重，劳累后加重，休息后减轻。重症肌无力 可以简单分成单纯眼肌型和全身型。从儿童到老年人都可能会发病，一般而言，20岁-40岁人群中，女 性患者多见 ...