[Pubmed] The best and worst of times in therapy development for myasthenia gravis

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Muscle Nerve. 2022 Nov 2. doi: 10.1002/mus.27742. Online ahead of print.

ABSTRACT

Within the last 5 years the U.S. Federal 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. Questions, however, about which patients are most likely to benefit from which therapies, and the relative effectiveness of these very expensive drugs, results 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 Myasthenia Gravis Foundation of America (MGFA) International Conference to discuss the most critical needs for clinical trial readiness and biomarker development in the context of therapy development for MG. The authors 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 publication. This article is protected by copyright. All rights reserved.

PMID:36321730 | DOI:10.1002/mus.27742


Source: https://pubmed.ncbi.nlm.nih.gov/3632173 ... 1&v=2.17.8