Eculizumab for the treatment of myasthenia gravis |
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| Authors: | Renato Mantegazza Paola Cavalcante |
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| Affiliation: | 1. Neurology IV Unit ? Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta , Milan, Italy renato.mantegazza@istituto-besta.it;3. Neurology IV Unit ? Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta , Milan, Italy |
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| Abstract: | ABSTRACT Introduction Acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG) is effectively treated with symptomatic and immunosuppressive drugs but a proportion of patients has a persistent disease and severe adverse events (AEs). The unmet medical needs are specific immunosuppression and AE lowering. Eculizumab blocks C5 protecting neuromuscular junction from the destructive autoantibody effects. Phase II (Study C08-001) and III (ECU-MG-301) studies, with the open-label extension (ECU-MG-302), demonstrated eculizumab efficacy and safety in refractory gMG patients. |
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| Keywords: | Biological drugs complement system eculizumab myasthenia gravis |
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