Generalized myasthenia gravis (gMG) is an autoimmune disease that causes disabling weakness via damage to the neuromuscular junction. In most patients, the disease is mediated by autoantibodies to the acetylcholine receptor, which activate the complement cascade. Our objective was to analyze response profiles in adult patients with anti-acetylcholine receptor antibody-positive refractory gMG treated with eculizumab—a terminal complement inhibitor—in the REGAIN study or its open-label extension (OLE).

中文翻译：

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依库珠单抗治疗难治性全身重症肌无力的长期疗效：反应者分析

全身性重症肌无力 (gMG) 是一种自身免疫性疾病，通过损伤神经肌肉接头导致致残性虚弱。在大多数患者中，这种疾病是由乙酰胆碱受体自身抗体介导的，乙酰胆碱受体激活补体级联反应。我们的目标是分析在 REGAIN 研究或其开放标签扩展 (OLE) 中使用 eculizumab（一种终末补体抑制剂）治疗的抗乙酰胆碱受体抗体阳性难治性 gMG 成年患者的反应概况。