筋疾患の治療の進歩

抄録

This article reviews the representative papers of treatment of muscular disorder published in 2017. There are many excellent works, which can provide a framework for the treatment of myasthenia gravis (MG). The mainstays of MG treatment are acetylcholinesterase inhibitors, and immunosuppressive and immunomodulatory therapies. MG presents many challenges for establishing treatment efficacy through clinical trials. Among these are the rarity and heterogeneity of the disease, spontaneous fluctuations, prolonged latency to effect for many immunosuppressive drugs, and the uncertain generalizability of results from randomized controlled trials. Prospective observational study designs may overcome some of these limitations, but attention is required for management for MG. In addition, we also present both basic and clinical investigation of inflammatory myopathies, Duchenne muscular dystrophy, and Pompe disease.