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

Get full text

Targeted therapy for tuberous sclerosis complex by E. D. Belousova, D. V. Vlodavets, A. M. Pivovarova, O. V. Katysheva, M. Yu. Dorofeeva

Get full text

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

Get full text

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

Get full text

Retrospective analysis of the natural history of type 1 spinal muscular atrophy in children by D. V. Vlodavets, N. V. Ganina, E. S. Ilina, A. A. Kokorina, L. M. Kuzenkova, E. Yu. Sapego, D. S. Smirnov, Yu. A. Shevtsova, A. V. Saulina, A. N. Petrov, N. A. Zolkin, Yu. N. Linkova, A. V. Zinkina-Orikhan

Get full text