Medstar Georgetown University Hospital, USA
Background: Myasthenia gravis (MG) is an autoimmune disorder of the neuromuscular junction mediated by antibodies against acetylcholine receptor (AChR), muscle-specific kinase (MuSK), and low-density lipoprotein receptor-related protein 4 (LRP4). Conventional therapies, including corticosteroids, immunosuppressants, intravenous immunoglobulin (IVIg), and plasma exchange, are effective but limited by delayed onset, incomplete response, and adverse effects. Advances in disease pathophysiology have led to the development of targeted biologic therapies.
Objective: To review recent advances in targeted therapies for MG, with
emphasis on clinical efficacy, safety, and their role in contemporary
management.
Methods: Key phase II and III clinical
trials and extension studies were reviewed, including REGAIN and CHAMPION-MG (complement inhibitors), and ADAPT, ADAPT+,
MycarinG, and VIVACITY-MG and VIVACITY-MG3 (FcRn antagonists).
Results: Complement inhibitors such as eculizumab (REGAIN) and ravulizumab
(CHAMPION-MG) significantly improve
clinical outcomes in refractory AChR-positive MG by inhibiting terminal complement activation. FcRn
antagonists, including efgartigimod (ADAPT, ADAPT+), rozanolixizumab
(MycarinG), and nipocalimab (VIVACITY-MG, VIVACITY-MG3), reduce circulating
pathogenic IgG levels and produce rapid, clinically meaningful improvements in
MG-ADL scores with favorable safety profiles. Emerging therapies targeting
B cells, including
inebilizumab, as well as plasma cells and cytokine pathways, are under investigation and may broaden treatment options,
particularly for refractory and seronegative MG.
Conclusion: Targeted therapies are reshaping MG management by providing faster and more effective disease control with improved tolerability. Future management will focus on selecting the right therapy for the right patient based on antibody profile, disease severity, and treatment response.
Dr.
Sara Dehbashi is board certified in Neurology, Neuromuscular medicine and
Electrodiagnostic medicine. She was graduated from Shiraz University of Medical
Sciences,completedaresidencyatUniversityofTexasMedicalBranchHospitalsanda
Neuromuscular fellowship atMountSinai Hospital.Dr.Dehbashi's main
clinical areas of interest are AIDP/CIDP, Myasthenia Gravis, Myopathies,
neuropathies, and motor neuron diseases. Dr. Dehbashi was previously affiliated
with Thomas Jefferson University Hospital and joined the Medstar Georgetown
University Hospital (MGUH) team in 2025 as an Associate Professor of
Neurology/Neuromuscular medicine. She is the Director of Myasthenia Gravis
Clinic at MGUH.