Affiliation:
1. Neuroimmunology and Neuromuscular Disease Department, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan
2. Department of Human Neurosciences Sapienza University of Rome, Piazzale Aldo Moro 5, 00185 Rome, Italy
Abstract
Purpose of reviewMyasthenia Gravis (MG) is a rare neurological disorder affecting the neuromuscular junction. Clinical hallmarks are fatigability and weakness affecting the extraocular, axial, limb and/or respiratory muscles. Despite immunosuppressive treatment, mainly based on corticosteroids and nonsteroidal immunosuppressants, the burden of MG is still significant, both in terms of inadequate disease control and burdensome side effects. Driven by such limits, the past years have been characterized by an escalation of MG drug development, with novel molecules which now focuses on having a more targeted effect, with a higher safety and efficacy profile.Recent findingsAs the pathogenic mechanism of MG are slowly being unravelled, new potential targets for treatments are being considered. This has led since 2017 to the Food and Drug Administration (FDA)-approval of three new drugs that either act by blocking the complement system (i.e., eculizumab and ravulizumab) or by blocking the neonatal Fc receptor thus preventing immunoglobulin recycling and reducing imunoglobulin G (IgG) antibodies (i.e., efgartigimod). Other drugs, with similar mechanism of action, are currently under review for approval.SummaryThe repertoire of available and developmental therapies for MG is rapidly expanding, finally responding to the unmet need of a more targeted and effective therapeutic approach in MG.
Publisher
Ovid Technologies (Wolters Kluwer Health)
Subject
Neurology (clinical),Neurology
Cited by
7 articles.
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