中国神经再生研究(英文版) ›› 2021, Vol. 21 ›› Issue (5): 1834-1848.doi: 10.4103/NRR.NRR-D-24-01011

• 综述:神经损伤修复保护与再生 • 上一篇    下一篇

重症肌无力的新疗法:从动物模型到临床应用的转化研究

  

  • 出版日期:2026-05-15 发布日期:2025-08-21

Novel therapies for myasthenia gravis: Translational research from animal models to clinical application

Benedetta Sorrenti1, 2, 3, Christian Laurini1, 2, 3, Luca Bosco1, 2, Camilla Mirella Maria Strano1, 2, 3, Adele Ratti1, 3, Yuri Matteo Falzone1, 2, Stefano Carlo Previtali1, 2, 3, *   

  1. 1 Neurology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;  2 Neuromuscular Repair Unit, Institute of Experimental Neurology (INSPE) and Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy;  3 Vita-Salute San Raffaele University, Milan, Italy
  • Online:2026-05-15 Published:2025-08-21
  • Contact: Stefano Carlo Previtali, MD, PhD, previtali.stefano@hsr.it.

摘要: https://orcid.org/0000-0003-2546-4357 (Stefano Carlo Previtali) 


Abstract: Myasthenia gravis is a chronic autoimmune disorder that affects the neuromuscular junction leading to fluctuating skeletal muscle fatigability. The majority of myasthenia gravis patients have detectable antibodies in their serum, targeting acetylcholine receptor, muscle-specific kinase, or related proteins. Current treatment for myasthenia gravis involves symptomatic therapy, immunosuppressive drugs such as corticosteroids, azathioprine, and mycophenolate mofetil, and thymectomy, which is primarily indicated in patients with thymoma or thymic hyperplasia. However, this condition continues to pose significant challenges including an unpredictable and variable disease progression, differing response to individual therapies, and substantial longterm side effects associated with standard treatments (including an increased risk of infections, osteoporosis, and diabetes), underscoring the necessity for a more personalized approach to treatment. Furthermore, about fifteen percent of patients, called “refractory myasthenia gravis patients”, do not respond adequately to standard therapies. In this context, the introduction of molecular therapies has marked a significant advance in myasthenia gravis management. Advances in understanding myasthenia gravis pathogenesis, especially the role of pathogenic antibodies, have driven the development of these biological drugs, which offer more selective, rapid, and safer alternatives to traditional immunosuppressants. This review aims to provide a comprehensive overview of emerging therapeutic strategies targeting specific immune pathways in myasthenia gravis, with a particular focus on preclinical evidence, therapeutic rationale, and clinical translation of B-cell depletion therapies, neonatal Fc receptor inhibitors, and complement inhibitors.

Key words: acetylcholine receptor (AChR), animal models, B-cell depletion, biological therapies, complement, immunotherapy, muscle-specific kinase (MuSK), neonatal Fc receptor