Sleep Disorders and Fatigue in Patients with Different Forms of Myotonic Dystrophy Type 1 von Erokhina, E. K., Melnik, E. A., Lebedeva, D. D., Shamtieva, K. V., Peters, T. V., Pavlikova, E. P., Gepard, V. V., Vlodavets, D. V.

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General principles of vaccination of patients with neuromuscular diseases von Skorikov, M. S., Vlodavets, D. V.

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Experience of using gene replacement therapy with Zolgensma® (onasemnogene abeparvovec) in real clinical practice in Russia von Artemyeva, S. B., Papina, Yu. O., Shidlovskaya, O. A., Monakhova, A. V., Vlodavets, D. V.

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SMArt Retro study: a retrospective data analysis of the Russian registry of patients with spinal muscular atrophy von Vlodavets, D. V., Shchagina, O. A., Polyakov, A. V., Kutsev, S. I.

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Clinical characteristics of different forms of myotonic dystrophy type 1 von Erokhina, E. K., Melnik, E. A., Vlodavets, D. V.

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Russian experience of DMD genetic database von Vlodavets, D, Reshetov, D, Artemieva, S, Litvinova, E, Shulyakova, I, Shidlovskaya, O, Kazakov, D, Belousova, E

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Modern methods of therapy of Duchenne muscular dystrophy: literature review with a clinical case von Artemyeva, S. B., Shidlovskaya, О. А., Papina, Yu. О., Monakhova, А. V., Shulyakov, I. V., Vlodavets, D. V.

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Cognitive and emotional disturbances in adult patients with myotonic dystrophy type 1 von Erokhina, E. K., Shamtieva, K. V., Melnik, E. A., Kazakov, D. O., Kurbatov, S. A., Pavlikova, E. P., Tikhonova, O. A., Mershina, E. A., Sinitsyn, V. E., Vlodavets, D. V.

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Approaches to pathogenetic therapy of spinal muscular atrophy in children and newborns von Artemyeva, S. B., Shidlovskaya, O. A., Papina, Yu. O., Monakhova, A. V., Shulyakova, I. V., Belousova, E. D., Germanenko, O. Yu, Vlodavets, D. V.

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Modification of the method for analysis of genome editing results using CRISPR/Cas9 system on preimplantation mouse embryos von Dimitireva, T. V., Reshetov, D. A., Zhernovkov, V. E., Vlodavets, D. E., Zotova, E. D., Ermolkevich, T. G., Deykin, A. V.

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The social-economic burden of spinal muscular atrophy in Russia von Kolbin, A. S., Vlodavets, D. V., Kurylev, A. A., Balykina, Yu. Ye, Proskurin, M. A., Mishinova, S. A., Germanenko, O. Yu, Kolbina, N. Yu

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The efficacy and safety of nusinersen within the expanded access program in Russia von Artemieva, S. B., Kuzenkova, L. M., Ilyina, E. S., Kursakova, Yu. A., Kolpakchi, L. M., Sapego, E. Yu, Golenko, A. A., Popovich, S. G., Parshin, D. V., Shidlovskaya, O. A., Papina, Yu. O., Monakhova, A. V., Vlodavets, D. V.

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21O RAINBOWFISH: 2-year efficacy and safety data of risdiplam in infants with presymptomatic SMA von Servais, L., Finkel, R., Farrar, M., Vlodavets, D., Zanoteli, E., Al-Muhaizea, M., Araújo, A., Nelson, L., Jaber, B., Gorni, K., Kletzl, H., Palfreeman, L., Gaki, E., Rabbia, M., Summers, D., Fontoura, P., Bertini, E.

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P212 RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA) von Finkel, R., Farrar, M., Servais, L., Vlodavets, D., Zanoteli, E., Al-Muhaizea, M., Prufer, A., Nelson, L., Fischer, C., Gerber, M., Gorni, K., Kletzl, H., Palfreeman, L., Gaki, E., Fontoura, P., Bertini, E.

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SMA - TREATMENT: EP.281 Branaplam in type 1 spinal muscular atrophy: second and third part of a phase II study von Jevtic, S., Carr, D., Dobrzycka-Ambrozewicz, A., Kotulzca-Jozwiak, K., Lvova, O., Pervunina, T., Petryaikina, Y., Shishimorov, I., Vlodavets, D., Nally, S., Ramos, H., Borowsky, B.

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SMA - TREATMENT von Jevtic, S., Carr, D., Dobrzycka-Ambrozewicz, A., Kotulzca-Jozwiak, K., Lvova, O., Pervunina, T., Petryaikina, Y., Shishimorov, I., Vlodavets, D., Nally, S., Ramos, H., Borowsky, B.

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FP.24 RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA) von Finkel, R., Farrar, M., Vlodavets, D., Zanoteli, E., Al-Muhaizea, M., Nelson, L., Prufer, A., Servais, L., Wang, Y., Fisher, C., Gerber, M., Gorni, K., Kletzl, H., Palfreeman, L., Scalco, R., Bertini, E.

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REGISTRIES AND CARE OF NEUROMUSCULAR DISORDERS von Artemyeva, S., Vlodavets, D., Monakhova, A., Melnik, E., Shulyakova, I., Shidlovskaya, O., Belousova, E., Reshetov, D.

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DMD TREATMENT: ANIMAL MODELS: P.203Exons 6 and 7 skipping test on new murine model of Duchenne muscular dystrophy von Egorova, T., Reshetov, D., Polikarpova, A., Vassilieva, S., Vlodavets, D., Deikin, A.

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REGISTRIES AND CARE OF NEUROMUSCULAR DISORDERS: P.280Registry of neuromuscular genetic disorders in Russia von Artemyeva, S., Vlodavets, D., Monakhova, A., Melnik, E., Shulyakova, I., Shidlovskaya, O., Belousova, E., Reshetov, D.

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