Unmet Needs in Myasthenia Gravis Care

Current myasthenia gravis treatment gaps center on seronegative and purely ocular disease populations, where limited therapeutic data restrict evidence-based treatment decisions. While FcRn inhibitors show emerging promise for seronegative patients, no FDA-approved therapies specifically target these populations, creating clinical management challenges. The lack of specific biomarkers for treatment selection represents another critical unmet need, as clinicians cannot predict which patients will respond to particular targeted therapies before initiating treatment.

Achievement of minimal symptom expression remains elusive for many patients despite available targeted therapies, with current treatments reaching MSE rates of only 45% to 50% rather than the desired 90% to 99% response rates. Even patients showing clinical improvement on robust complement inhibitors or FcRn inhibitors frequently fail to achieve complete symptom resolution, indicating need for additional therapeutic mechanisms or combination approaches. This limitation affects quality-of-life and functional restoration goals that have become standard treatment objectives.

The development of reliable therapeutic biomarkers represents the most critical need for advancing precision medicine in myasthenia gravis. Such biomarkers would enable clinicians to predict treatment responses, optimize therapeutic selection, and potentially identify patients requiring combination therapies targeting multiple pathophysiological mechanisms. Without these predictive tools, treatment remains largely empirical, potentially delaying optimal therapy and prolonging patient disability. Future research must prioritize biomarker discovery alongside novel therapeutic development to achieve personalized medicine goals and improve treatment efficiency for all myasthenia gravis patient populations.