重症肌无力
医学
伊库利珠单抗
乙酰胆碱受体
自身抗体
临床试验
耐火材料(行星科学)
疾病
受体
内科学
免疫学
抗体
补体系统
天体生物学
物理
作者
Renato Mantegazza,Paola Cavalcante
出处
期刊:Current Opinion in Rheumatology
[Ovid Technologies (Wolters Kluwer)]
日期:2019-11-01
卷期号:31 (6): 623-633
被引量:38
标识
DOI:10.1097/bor.0000000000000647
摘要
Purpose of review This article provides an update on the most recent advances in diagnostic procedures and therapeutic approaches for myasthenia gravis, spanning from autoantibody and neuroelectrophysiological tests as diagnostic tools, to innovative and promising treatments based on biological drugs. Recent findings Novel studies performed by cell-based assays (CBAs) indicate an improvement in the chance of identifying serum autoantibodies in myasthenic patients. Clinical trials on the use of biological drugs were recently concluded, providing important data on safety and efficacy of eculizumab, efgartigimod and amifampridine phosphate: the first, a complement blocker, showed long-term safety and efficacy in acetylcholine receptor (AChR)-positive myasthenic patients with refractory generalized disease; the second, the neonatal Fc receptor blocker, was well tolerated and clinically effective in both AChR-specific and muscle-specific kinase receptor (MuSK)-positive patients; the third, a blocker of presynaptic potassium channels, was found to be well tolerated and effective in MuSK-positive patients. Summary CBAs can lead to a significant reduction of seronegative patients, improving myasthenia gravis diagnostic process. New biological drugs offer innovative approaches to treat myasthenic patients with generalized disease, promising to change the paradigm of treatment and to significantly enhance therapeutic success within a precision medicine framework.
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