The efficacy and safety of nusinersen within the expanded access program in Russia

The efficacy and safety of nusinersen within the expanded access program in Russia

Introduction. Spinal muscular atrophy is a severe neuromuscular disease characterized by rapid progression of muscle weakness and early death. Pathogenetic therapy with nusinersen can significantly change the course of the disease and enable the patient to acquire new skills. The study of the effica...

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Main Authors: S. B. Artemieva, L. M. Kuzenkova, E. S. Ilyina, Yu. A. Kursakova, L. M. Kolpakchi, E. Yu. Sapego, A. A. Golenko, S. G. Popovich, D. V. Parshin, O. A. Shidlovskaya, Yu. O. Papina, A. V. Monakhova, D. V. Vlodavets
Format: Article
Language:Russian
Published: ABV-press 2020-12-01
Series:Нервно-мышечные болезни
Subjects:
spinal muscular atrophy
nusinersen
expanded access program
smn1 gene
Online Access:https://nmb.abvpress.ru/jour/article/view/396
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