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CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human DMD gene

Disease models & mechanisms, 2019-04, Vol.12 (4)

2019

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Experience of using gene replacement therapy with Zolgensma® (onasemnogene abeparvovec) in real clinical practice in Russia

Nervno-myshechnye bolezni, 2022, Vol.12 (1), p.29-38

2022

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Duchenne Muscular Dystrophy: Modern Approaches in Patient Management

Pediatricheskai͡a︡ farmakologii͡a︡ : nauchno-prakticheskiĭ zhurnal Soi͡u︡za pediatrov Rossii, 2023-11, Vol.20 (5), p.427-453

2023

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Sleep Disorders and Fatigue in Patients with Different Forms of Myotonic Dystrophy Type 1

Neuroscience and behavioral physiology, 2024, Vol.54 (1), p.35-40

2024

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Health Technologies Assessment for Orphan Diseases. Example of Social and Economic Burden of Spinal Muscular Atrophy

Pediatricheskai͡a︡ farmakologii͡a︡ : nauchno-prakticheskiĭ zhurnal Soi͡u︡za pediatrov Rossii, 2021-12, Vol.18 (5), p.408-415

2021

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Risdiplam for the treatment of spinal muscular atrophy

Zhurnal nevrologii i psikhiatrii imeni S.S. Korsakova, 2024, Vol.124 (2), p.45

Vlodavets, D V

2024

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The social-economic burden of spinal muscular atrophy in Russia

Farmakoèkonomika (Moskva. Online), 2021-02, Vol.13 (4), p.337-354

2021

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

Nervno-myshechnye bolezni, 2020-12, Vol.10 (3), p.35-41

2020

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General principles of vaccination of patients with neuromuscular diseases

Nervno-myshechnye bolezni, 2023-10, Vol.13 (3), p.40-47

2023

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The functional state of the respiratory system of the patients with proximal spinal muscular atrophy 5q (SMA 5q): from the natural history of the disease to the era of pathogenetic therapy. Problems and expectations

Rossiĭskiĭ vestnik perinatologii i pediatrii, 2024-08, Vol.69 (4), p.16-30

2024

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Clinical characteristics of different forms of myotonic dystrophy type 1

Russkiĭ zhurnal detskoĭ nevrologii, 2023-06, Vol.18 (1), p.22-37

2023

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SMArt Retro study: a retrospective data analysis of the Russian registry of patients with spinal muscular atrophy

Nervno-myshechnye bolezni, 2024-09, Vol.14 (3), p.54-71

2024

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Sleep disorders and fatigue in patients with different forms of myotonic dystrophy type 1

Zhurnal nevrologii i psikhiatrii imeni S.S. Korsakova, 2023, Vol.123 (8), p.62

2023

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Modern methods of therapy of Duchenne muscular dystrophy: literature review with a clinical case

Nervno-myshechnye bolezni, 2024-01, Vol.13 (4), p.103-112

2024

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Approaches to pathogenetic therapy of spinal muscular atrophy in children and newborns

Nervno-myshechnye bolezni, 2024-03, Vol.14 (1), p.86-92

2024

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Cognitive and emotional disturbances in adult patients with myotonic dystrophy type 1

Nervno-myshechnye bolezni, 2024-05, Vol.14 (2), p.12-24

2024

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Retrospective analysis of the natural history of type 1 spinal muscular atrophy in children

Rossiĭskiĭ vestnik perinatologii i pediatrii, 2021-09, Vol.66 (4), p.64-73

2021

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DIAGNOSTIC OPPORTUNITIES OF MUSCLE MRI IN NEUROMUSCULAR DISORDERS

Nevrologicheskiĭ zhurnal = Journal of neurology, 2015-07, Vol.19 (3), p.4

2015

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Modification of the method for analysis of genome editing results using CRISPR/Cas9 system on preimplantation mouse embryos

Bulletin of Russian State Medical University, 2016-05 (3), p.15-20

2016

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Targeted therapy for tuberous sclerosis complex

Rossiĭskiĭ vestnik perinatologii i pediatrii, 2016-01, Vol.61 (5), p.106-112

2016

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Analysis of phenotype expressions of deletions in the dystrophin gene in terms of efficiency of exon skipping as a method for treatment of hereditary dystrophinopathies

Bulletin of Russian State Medical University, 2016-05 (3), p.21-26

2016

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L.O. Badalyan and current progress in the study of hereditary neuromuscular diseases

L.O. Badalyan Neurological Journal, 2020-04, Vol.1 (1), p.64-72

2020

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Asymptomatic troponin I elevation and acute myocarditis in the late period of gene replacement therapy with onasemnogene aberpavovec for patients with spinal muscular atrophy: literature review and clinical case series

L.O. Badalyan Neurological Journal, 2023-10, Vol.4 (3), p.144-157

2023

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Expert council resolution on the use of pathogenetic therapy with Ataluren in patients with non-ambulatory Duchenne muscular dystrophy

L.O. Badalyan Neurological Journal, 2023-01, Vol.3 (4), p.148-151

2023

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Hospital aspergillosis

Žurnal mikrobiologii, ėpidemiologii i immunobiologii, 1984-06 (6), p.9

Vlodavets, V V

1984

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CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human DMD gene

Experience of using gene replacement therapy with Zolgensma® (onasemnogene abeparvovec) in real clinical practice in Russia

Duchenne Muscular Dystrophy: Modern Approaches in Patient Management

Sleep Disorders and Fatigue in Patients with Different Forms of Myotonic Dystrophy Type 1

Health Technologies Assessment for Orphan Diseases. Example of Social and Economic Burden of Spinal Muscular Atrophy

Risdiplam for the treatment of spinal muscular atrophy

The social-economic burden of spinal muscular atrophy in Russia

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

General principles of vaccination of patients with neuromuscular diseases

The functional state of the respiratory system of the patients with proximal spinal muscular atrophy 5q (SMA 5q): from the natural history of the disease to the era of pathogenetic therapy. Problems and expectations

Clinical characteristics of different forms of myotonic dystrophy type 1

SMArt Retro study: a retrospective data analysis of the Russian registry of patients with spinal muscular atrophy

Sleep disorders and fatigue in patients with different forms of myotonic dystrophy type 1

Modern methods of therapy of Duchenne muscular dystrophy: literature review with a clinical case

Approaches to pathogenetic therapy of spinal muscular atrophy in children and newborns

Cognitive and emotional disturbances in adult patients with myotonic dystrophy type 1

Retrospective analysis of the natural history of type 1 spinal muscular atrophy in children

DIAGNOSTIC OPPORTUNITIES OF MUSCLE MRI IN NEUROMUSCULAR DISORDERS

Modification of the method for analysis of genome editing results using CRISPR/Cas9 system on preimplantation mouse embryos

Targeted therapy for tuberous sclerosis complex

Analysis of phenotype expressions of deletions in the dystrophin gene in terms of efficiency of exon skipping as a method for treatment of hereditary dystrophinopathies

L.O. Badalyan and current progress in the study of hereditary neuromuscular diseases

Asymptomatic troponin I elevation and acute myocarditis in the late period of gene replacement therapy with onasemnogene aberpavovec for patients with spinal muscular atrophy: literature review and clinical case series

Expert council resolution on the use of pathogenetic therapy with Ataluren in patients with non-ambulatory Duchenne muscular dystrophy

Hospital aspergillosis

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