Evolving Landscape of Myasthenia Gravis Treatment and Unmet Needs: Leila Darki, MD

WATCH TIME: 4 minutes | Captions are auto-generated and may contain errors.

"I would like to highlight the trend of focusing more on finding biological markers, like biomarkers for [myasthenia gravis], predicting response to treatment, and also assessing the response throughout treatment. The goal is to individualize treatment based on these biomarkers and anticipate which treatment might work better for each individual patient."

Treatment of generalized myasthenia gravis (gMG) has traditionally involved the use of broad immunosuppressive agents to mitigate antibody-mediated injury at the neuromuscular junction. Although these therapies have demonstrated efficacy, their use may be associated with potential adverse effects. More recently, targeted approaches such as FcRn antagonists and complement inhibitors have been introduced, with the aim of providing comparable efficacy while potentially reducing systemic risks. Current evidence has indicated that these agents are generally well-tolerated, and that their introduction has contributed to changes in the therapeutic approach to gMG.¹

At the 2025 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) meeting, held October 29 to November 1, in San Francisco, California, neurologist Leila Darki, MD, presented on targeted treatments in gMG, specifically on zilucoplan (Zilbrysq; UCB Pharma) in a sponsored product theater.² Darki, an associate professor of clinical neurology at Keck Medicine of University of South Carolina, spoke with CGTLive®, our sister publication, at the meeting to highlight the progress being made in MG treatment, including novel targeted and cellular therapies such as CAR-T, and deepen clinician's understanding of the disease pathophysiology.

During the interview, she emphasized the emerging focus on identifying reliable biomarkers to predict and monitor treatment response, which could potentially help clinicians better individualize care for patients with gMG. Despite these advancements, she noted persistent gaps in care, particularly for patients with ocular or seronegative MG, and the continued difficulty in determining optimal treatment pathways. Further into the conversation, Darki underscored the importance of capturing patients’ day-to-day challenges, which often go beyond what is measured during clinical visits, in order to better address their needs.

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REFERENCES
1. Wolfe GI, Hanson JE, Silvestri NJ. Myasthenia gravis: The evolving therapeutic landscape. eNeurologicalSci. 2024;37:100541. Published 2024 Nov 19. doi:10.1016/j.ensci.2024.100541
2. Darki, L. Zilbrysq Featured Presentation: C5 Targeted Treatment. Presented at: 2025 AANEM; October 29 to November 1; San Francisco, California. Presentation Stage by UCB, Inc.