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Published on: October 25, 2018
New Targeted Agents in Myasthenia Gravis and Future Therapeutic Strategies
Daniel Sánchez-Tejerina1, Javier Sotoca1, Arnau Llaurado1
1Clinic of Neuromuscular Disorders and Rare Diseases, Neurology Department, Hospital Universitari Vall d'Hebron, Vall d'Hebron Research Institute, European Reference Network for Neuromuscular and Rare Diseases EURO-NMD, 08035 Barcelona, Spain.
Abstract:
Myasthenia gravis (MG) is a chronic autoimmune disease for which multiple immunomodulatory therapies are available. Nevertheless, MG has a significant impact on patient quality of life. In recent years, experts' main efforts have focused on optimizing treatment strategies, since disease burden is considerably affected by their safety and tolerability profiles, especially in patients with refractory phenotypes. This article aims to offer neurologists caring for MG patients an overview of the most innovative targeted drugs specifically designed for this disease and summarizes the recent literature and more recent evidence on agents targeting B cells and plasmablasts, complement inhibitors, and neonatal fragment crystallizable receptor (FcRn) antagonists. Positive clinical trial results have been reported, and other studies are ongoing. Finally, we briefly discuss how the introduction of these novel targeted immunological therapies in a changing management paradigm would affect not only clinical outcomes, disease burden, safety, and tolerability, but also health spending in a condition that is increasingly managed based on a patient-centred model.
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