The best and worst of times in therapy development for myasthenia gravis.
The best and worst of times in therapy development for myasthenia gravis.
复制标题
重症肌无力治疗发展的最佳和最差时期。
DOI：
10.1002/mus.27742
复制
发表时间：
2023
期刊：
影响因子：
3.4
通讯作者：
Kaminski,HenryJ
中科院分区：
文献类型：
--
作者：
Benatar,Michael;Cutter,Gary;Kaminski,HenryJ
Within the last 5 years, the US Food and Drug Administration (FDA) has approved complement and neonatal Fc receptor (FcRN) inhibitors for treatment of generalized myasthenia gravis, and several other therapies are in late‐stage clinical trials or under regulatory review. However, questions about which patients are most likely to benefit from which therapies, and the relative effectiveness of these very expensive drugs, has resulted in uncertainty around the place that they should occupy in the existing therapeutic armamentarium. MGNet (a Rare Diseases Clinical Research Consortium funded by the National Institute of Neurological Diseases and Stroke) held two meetings during the 14th International Conference of the Myasthenia Gravis Foundation of America to discuss the most critical needs for clinical trial readiness and biomarker development in the context of therapy development for myasthenia gravis. Herein we provide a summary of these discussions, but not a consensus opinion, and offer a series of recommendations to guide focused research in the most critical areas. We welcome ongoing discussion through comments on this work.
