Myasthenia gravis (MG) is an autoimmune disorder that affects the neuromuscular junction, leading to muscle weakness and fatigue. MG is caused by antibodies against the acetylcholine receptor (AChR), the muscle-specific kinase (MuSK) or other AChR-related proteins that are expressed in the postsynaptic muscle membrane. The standard therapeutic approach for MG has relied on acetylcholinesterase inhibitors, corticosteroids and immunosuppressants, which have shown good efficacy in improving MG-related symptoms in most people with the disease; however, these therapies can carry a considerable burden of long-term adverse effects. Moreover, up to 15% of individuals with MG exhibit limited or no response to these standard therapies. The emergence of molecular therapies, including monoclonal antibodies, B cell-depleting agents and chimeric antigen receptor T cell-based therapies, has the potential to revolutionize the MG treatment landscape. This Review provides a comprehensive overview of the progress achieved in molecular therapies for MG associated with AChR antibodies and MuSK antibodies, elucidating both the challenges and the opportunities these therapies present to the field. The latest developments in MG treatment are described, exploring the potential for personalized medicine approaches.

重症肌无力 (MG) 是一种自身免疫性疾病，会影响神经肌肉接头，导致肌肉无力和疲劳。MG 是由抗乙酰胆碱受体 (AChR)、肌肉特异性激酶 (MuSK) 或突触后肌膜中表达的其他 AChR 相关蛋白的抗体引起的。MG 的标准治疗方法依赖于乙酰胆碱酯酶抑制剂、皮质类固醇和免疫抑制剂，这些药物在改善大多数 MG 患者的相关症状方面显示出良好的疗效；然而，这些疗法可能会带来相当大的长期不良反应负担。此外，高达 15% 的 MG 患者对这些标准疗法的反应有限或没有反应。分子疗法的出现，包括单克隆抗体、B 细胞耗竭剂和基于嵌合抗原受体 T 细胞的疗法，有可能彻底改变 MG 治疗格局。本综述全面概述了与 AChR 抗体和 MuSK 抗体相关的 MG 分子疗法所取得的进展，阐明了这些疗法给该领域带来的挑战和机遇。描述了 MG 治疗的最新进展，探索了个性化医疗方法的潜力。