Research Progress in Pharmacological Targeted Therapy for Myasthenia Gravis

Myasthenia gravis (MG) is a disorder of neuromuscular junction transmission that is mediated by autoantibodies and depends on T cell help, causing fatigable muscle weakness. Limitations of conventional first-line treatments, including long-term adverse effects and inadequate control of refractory disease, have driven targeted therapy to the forefront of MG research. This narrative review summarizes second-line and later-line targeted biological and cellular therapies, covering agents directedat B and T cells, complement inhibitors, neonatal Fc receptor (FcRn) antagonists and cytokine inhibitors, with emphasis on mechanisms, efficacy, safety and regulatory status. Several key knowledge gaps remain, particularly around treatment positioning, evidence tailored to disease subtypes and long-term safety. The most recent pivotal trial and real-world evidence is incorporated, and key priorities for future research are outlined.