K. D. Popov, T. M. Alekseeva, V. D. Nazarov, A. I. Vlasenko, S. M. Malyshev
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引用次数: 0
Abstract
Currently, there are three drugs in the world for the pathogenetic therapy of spinal muscular atrophy 5q: nusinersen, risdiplam and onasemnogene abeparvovek. At the same time, it is still unknown to what extent this treatment is able to change the natural history of the disease, and the development of methods for evaluating the effectiveness of treatment is the subject of active scientific research. This article is a review of studies of laboratory approaches for assessing the disease severity and the response to nusinersen therapy in patients with spinal muscular atrophy 5q in various age groups.
期刊介绍：
The principal objective of the "Neuromuscular Diseases" journal is publication of state-of-art information about scientific clinical studies, diagnostics, and treatment of neurological diseases. "Neuromuscular Diseases" is a peer-reviewed journal, and members of its editorial board are major professionals in Russia and the CIS countries that have deserved authority not only in our country but abroad as well. Besides, leading scientists of Europe and the USA regularly cooperate with the journal. The journal regularly publishes reviews of modern literature references, proprietary clinical observations, lectures, original articles, results of research protocols in various areas of neuromuscular pathology - clinical presentation, diagnostics, medication, surgical and non-drug therapies, modern trends in drug rehabilitation, and recent drug developments. Due to international cooperation, the journal contains the newest therapeutic and diagnostic approaches to monitoring of patients with neuromuscular pathology.
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