Future perspectives on myasthenia gravis and related disorders.

International review of neurobiology Pub Date : 2025-01-01 Epub Date: 2025-05-09 DOI:10.1016/bs.irn.2025.04.016
Carolina Barnett Tapia, Anna Rostedt Punga
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Abstract

The diagnostic precision of MG, along with the emergence of novel treatments targeting the autoimmune response, has ushered the field into a new era, moving MG management closer to personalized medicine. However, critical gaps remain, including the absence of approved treatments for seronegative MG, limited attention to Lambert-Eaton myasthenic syndrome and congenital myasthenic syndromes, and the need for more precise biomarkers. Current clinical trials primarily rely on outcomes that measure symptoms and/or muscle weakness/fatigability; however, there is a need for biomarkers that better reflect disease activity, enable early diagnosis in seronegative MG, and identify the underlying antibodies in these patients. Predictive biomarkers are also needed to assess the risk of generalization from ocular MG and the likelihood of relapses. Furthermore, despite their efficacy, novel treatments such as complement and FcRn inhibitors are costly and inaccessible in many countries. Future MG research must, therefore, prioritize socioeconomic considerations alongside therapeutic advancements. Also, a better understanding of the fatigue in MG, with differences in men and women, is essential to better design treatment over time.

重症肌无力及其相关疾病的未来展望。
MG的诊断精度,以及针对自身免疫反应的新治疗方法的出现,将该领域带入了一个新时代,使MG的管理更接近个性化医疗。然而,关键的空白仍然存在,包括缺乏批准的血清阴性MG治疗方法,对Lambert-Eaton肌无力综合征和先天性肌无力综合征的关注有限,以及需要更精确的生物标志物。目前的临床试验主要依赖于测量症状和/或肌肉无力/疲劳的结果;然而,需要更好地反映疾病活动的生物标志物,能够在血清阴性MG患者中进行早期诊断,并识别这些患者的潜在抗体。还需要预测性生物标志物来评估眼部MG的泛化风险和复发的可能性。此外,尽管补体和FcRn抑制剂等新型治疗方法有效,但在许多国家价格昂贵且难以获得。因此,未来的MG研究必须在治疗进展的同时优先考虑社会经济因素。此外,更好地了解MG患者的疲劳,以及男性和女性的差异,对于更好地设计治疗方案至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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