Molecular markers of disease severity and response to nusinersen therapy in 5q spinal muscular atrophy (literature review)

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...

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Bibliographic Details
Main Authors: K. D. Popov, T. M. Alekseeva, V. D. Nazarov, A. I. Vlasenko, S. M. Malyshev
Format: Article
Language:Russian
Published: ABV-press 2023-10-01
Series:Нервно-мышечные болезни
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Online Access:https://nmb.abvpress.ru/jour/article/view/557
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