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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| Format: | Article |
| Language: | Russian |
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ABV-press
2023-10-01
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| Series: | Нервно-мышечные болезни |
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| Online Access: | https://nmb.abvpress.ru/jour/article/view/557 |
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| author | K. D. Popov T. M. Alekseeva V. D. Nazarov A. I. Vlasenko S. M. Malyshev |
| author_facet | K. D. Popov T. M. Alekseeva V. D. Nazarov A. I. Vlasenko S. M. Malyshev |
| author_sort | K. D. Popov |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-ee4d26c3f9ae4d4dbe026e39f3555988 |
| institution | Kabale University |
| issn | 2222-8721 2413-0443 |
| language | Russian |
| publishDate | 2023-10-01 |
| publisher | ABV-press |
| record_format | Article |
| series | Нервно-мышечные болезни |
| spelling | doaj-art-ee4d26c3f9ae4d4dbe026e39f35559882025-08-20T03:38:15ZrusABV-pressНервно-мышечные болезни2222-87212413-04432023-10-01133333910.17650/2222-8721-2023-13-3-33-39355Molecular markers of disease severity and response to nusinersen therapy in 5q spinal muscular atrophy (literature review)K. D. Popov0T. M. Alekseeva1V. D. Nazarov2A. I. Vlasenko3S. M. Malyshev4V.A. Almazov National Medical Research Center, Ministry of Health of RussiaV.A. Almazov National Medical Research Center, Ministry of Health of RussiaI.P. Pavlov First Saint Petersburg State Medical UniversityV.A. Almazov National Medical Research Center, Ministry of Health of RussiaV.A. Almazov National Medical Research Center, Ministry of Health of RussiaCurrently, 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.https://nmb.abvpress.ru/jour/article/view/557spinal muscular atrophymarkers of treatment responsenusinersenneurofilamentstau-protein |
| spellingShingle | K. D. Popov T. M. Alekseeva V. D. Nazarov A. I. Vlasenko S. M. Malyshev Molecular markers of disease severity and response to nusinersen therapy in 5q spinal muscular atrophy (literature review) Нервно-мышечные болезни spinal muscular atrophy markers of treatment response nusinersen neurofilaments tau-protein |
| title | Molecular markers of disease severity and response to nusinersen therapy in 5q spinal muscular atrophy (literature review) |
| title_full | Molecular markers of disease severity and response to nusinersen therapy in 5q spinal muscular atrophy (literature review) |
| title_fullStr | Molecular markers of disease severity and response to nusinersen therapy in 5q spinal muscular atrophy (literature review) |
| title_full_unstemmed | Molecular markers of disease severity and response to nusinersen therapy in 5q spinal muscular atrophy (literature review) |
| title_short | Molecular markers of disease severity and response to nusinersen therapy in 5q spinal muscular atrophy (literature review) |
| title_sort | molecular markers of disease severity and response to nusinersen therapy in 5q spinal muscular atrophy literature review |
| topic | spinal muscular atrophy markers of treatment response nusinersen neurofilaments tau-protein |
| url | https://nmb.abvpress.ru/jour/article/view/557 |
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