Muppidi S, et al. - Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN), researchers reported an interim analysis of an open-label extension of REGAIN, assessing the long-term safety and effectiveness of eculizumab. One hundred seventeen patients received eculizumab (1,200 mg every 2 weeks for 22.7 months [median]). Findings revealed that eculizumab's safety profile was compatible with REGAIN. During the interim analysis period, no cases of meningococcal infection were reported. From the year before REGAIN, myasthenia gravis exacerbation rate was decreased by 75%. In REGAIN, improvements with eculizumab in daily living activities, muscle strength, functional ability, and quality of life have been maintained for 3 years. Data reported that 56% of patients achieved minimal manifestations or pharmacological remission. During open-label eculizumab, patients receiving placebo during REGAIN experienced fast and sustained improvements. These findings demonstrate the long-term safety and sustained effectiveness of eculizumab for refractory gMG.