Efficacy and Safety of Gefurulimab in Generalized Myasthenia Gravis

Is gefurulimab a relatively safe and efficacious treatment for adults with anti–acetylcholine receptor antibody–positive (AChR-Ab+) generalized myasthenia gravis (gMG)? In this randomized clinical trial in AChR-Ab+ gMG, gefurulimab treatment reduced Myasthenia Gravis Activities of Daily Living total score at week 26 compared with placebo with statistically significant treatment effect, achieving the primary end point for the study. The incidence of adverse events was similar between treatment groups. Gefurulimab, a novel dual-binding nanobody administered via weekly subcutaneous self-injection, demonstrated the potential to offer adults with AChR-Ab+ gMG an efficacious and convenient treatment option.

Corresponding Author: Kelly G. Gwathmey, MD, CUIMC/Neurological Institute of New York, 710 W 168th St, New York, NY 10032 (kgg2125@cumc.columbia.edu).

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