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Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study
Journal article   Peer reviewed

Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study

James F Howard Jr, Kimiaki Utsugisawa, Michael Benatar, Hiroyuki Murai, Richard J Barohn, Isabel Illa, Saiju Jacob, John Vissing, Ted M Burns, John T Kissel, …
Lancet neurology, Vol.16(12), pp.976-986
12/2017
DOI: 10.1016/S1474-4422(17)30369-1
PMID: 29066163

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Abstract

Complement is likely to have a role in refractory generalised myasthenia gravis, but no approved therapies specifically target this system. Results from a phase 2 study suggested that eculizumab, a terminal complement inhibitor, produced clinically meaningful improvements in patients with anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis. We further assessed the efficacy and safety of eculizumab in this patient population in a phase 3 trial.
Drug Resistance Severity of Illness Index Antibodies, Monoclonal, Humanized - adverse effects Double-Blind Method Myasthenia Gravis - blood Outcome Assessment (Health Care) Autoantibodies - blood Humans Middle Aged Male Antibodies, Monoclonal, Humanized - administration & dosage Myasthenia Gravis - physiopathology Antibodies, Monoclonal, Humanized - pharmacology Adult Female Receptors, Cholinergic - immunology Aged Myasthenia Gravis - drug therapy

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