{"title":"重症肌无力及其相关疾病的未来展望。","authors":"Carolina Barnett Tapia, Anna Rostedt Punga","doi":"10.1016/bs.irn.2025.04.016","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":94058,"journal":{"name":"International review of neurobiology","volume":"183 ","pages":"191-197"},"PeriodicalIF":0.0000,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Future perspectives on myasthenia gravis and related disorders.\",\"authors\":\"Carolina Barnett Tapia, Anna Rostedt Punga\",\"doi\":\"10.1016/bs.irn.2025.04.016\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":94058,\"journal\":{\"name\":\"International review of neurobiology\",\"volume\":\"183 \",\"pages\":\"191-197\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"International review of neurobiology\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1016/bs.irn.2025.04.016\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/5/9 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"International review of neurobiology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/bs.irn.2025.04.016","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/5/9 0:00:00","PubModel":"Epub","JCR":"","JCRName":"","Score":null,"Total":0}
Future perspectives on myasthenia gravis and related disorders.
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.