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pro vyhledávání: '"Alena V. Naidenko"'
Autor:
Lyudmila M. Kuzenkova, Evgeniya V. Uvakina, Sofiya G. Popovich, Tatyana V. Podkletnova, Aleksandra A. Nezhelskaya, Luizat M. Abdullaeva, Darya A. Fisenko, Alena V. Naidenko
Publikováno v:
Russian Pediatric Journal. 25:18-22
Goal. To evaluate the safety profile of onasemnogene abeparvovec (Zolgensma®) gene therapy in children with spinal muscular atrophy (SMA) in real clinical practice. Materials and methods. The study included 30 SMA children who underwent gene replace