Background and Objectives: Ravulizumab, a long-acting C5 complement inhibitor, was approved in the US and Europe in 2022 as an add-on therapy for the standard treatment of AChR-positive generalized MG (gMG). We share our real-world experience with adult patients receiving this therapy in Romania. Materials and Methods: Six AChR-positive gMG patients received ravulizumab through an Early Access Program (January 2023–October 2024). Patient outcomes were assessed at the therapy start and q8w using Quantitative MG (QMG), MG Activities of Daily Living (MG-ADL), and MG Quality of Life 15-item revised (MG-QoL15r) scales. Results: Age at disease onset ranged from 15 to 35 years. Four of the six patients were women. Two patients had gMG severity level of IIa, and four patients of IIb according to the Myasthenia Gravis Foundation of America (MGFA) classification. Five patients experienced rapid and sustained improvements in MG symptoms with MG-ADL score reductions ranged from −3 to −5 at 26 weeks post-ravulizumab start (except for those with a low baseline score: three and one). QMG score dropped in three patients (−2 to 12) during the treatment period, increased in two (+2 and +8), and remained stable in one (zero). Three patients showed sustained improvement in MG symptoms after ≥60 weeks. MG-QoL15r significantly dropped (−22 to −10) throughout the treatment period. One patient experienced ravulizumab-associated adverse events (vomiting, diarrhea, chills) that resolved within 24 h following symptomatic management, two to three episodes of myasthenic exacerbations during treatment, and discontinued it. Conclusions: All cases presented here had early-onset AChR antibody-positive, non-thymomatous MG. Despite differences in disease duration and underlying conditions, clinically meaningful and sustained improvements in gMG symptoms, and reduced corticosteroid doses were observed in all patients except one after adding ravulizumab to the treatment plan.

Background: Efgartigimod alfa, the first neonatal Fc receptor inhibitor approved for the treatment of acetylcholine receptor antibody (AChR)-positive generalized myasthenia gravis (MG), has demonstrated efficacy in randomized controlled trials. However, real-world data, particularly from Eastern Europe, remain limited. Methods: This prospective, multicenter study enrolled AChR-positive generalized MG patients treated with efgartigimod across six Romanian university hospitals between March 2024 and September 2025. Outcomes were assessed using the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale, the Quantitative Myasthenia Gravis (QMG) score, and the Myasthenia Gravis Quality of Life 15-item revised scale (MG-QoL15r). Clinically meaningful improvement (CMI), minimal symptom expression (MSE), patient-acceptable symptom state (PASS), subdomain response, and corticosteroid sparing were evaluated. Results: Thirty-nine patients completed at least one treatment cycle, receiving a total of 210 cycles. In cycle 1, CMI rates were 89.7% for MG-ADL and 74.4% for QMG, with a median MG-ADL reduction of 4.0 points ( p < 0.001); 69.2% achieved CMI after a single infusion. Composite PASS increased from 25.6% in cycle 1 to 54.5% in cycle 8. Subdomain analysis showed improvements across all domains for both the MG-ADL and the QMG, with the exception of QMG respiratory. Among patients on corticosteroids ( n = 29), median prednisone dose decreased from 20.0 to 10.0 mg/day ( p = 0.004), with 58.6% achieving reduction or discontinuation. Conclusion: This first Eastern European real-world efgartigimod-treated cohort confirms rapid and sustained improvement and corticosteroid sparing, consistent with prior evidence.