Results of a two-year comprehensive follow-up in patients with spinal muscular atrophy who received gene therapy with onasemnogen abeparvovec at the presymptomatic stage of the disease

Lyudmila M. Kuzenkova; Aleksey L. Kurenkov; Eugeniya V. Uvakina; Daria A. Fisenko; Vladislav V. Chernikov; Elena L. Semikina; Sofia G. Popovich; Bella I. Bursagova; Oxana V. Globa; Natalya V. Andreenko; Luizat M. Abdullaeva; Julia A. Kurova; Nadezhda S. Adalimova; Daria S. Nikolenko; Anastasia A. Lyalina; Olga V. Komarova

L.O. Badalyan Neurological Journal
Nevrologičeskij žurnal imeni L.O. Badalâna

ISSN 2686-8997 (Print)
ISSN 2712-794X (Online)

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Kuzenkova L.M., Kurenkov A.L., Uvakina E.V., Fisenko D.A., Chernikov V.V., Semikina E.L., Popovich S.G., Bursagova B.I., Globa O.V., Andreenko N.V., Abdullaeva L.M., Kurova J.A., Adalimova N.S., Nikolenko D.S., Lyalina A.A., Komarova O.V. Results of a two-year comprehensive follow-up in patients with spinal muscular atrophy who received gene therapy with onasemnogen abeparvovec at the presymptomatic stage of the disease. L.O. Badalyan Neurological Journal. 2025;6(4):182-194. (In Russ.) https://doi.org/10.46563/2686-8997-2025-6-4-182-194. EDN: bletts

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ISSN 2686-8997 (Print)
ISSN 2712-794X (Online)

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Lyudmila M. Kuzenkova

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About the Authors

Lyudmila M. Kuzenkova
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation; N.F. Filatov Clinical Institute of Children’s Health, I.M. Sechenov First Moscow State Medical University (Sechenov University), Moscow, 119435, Russian Federation
Russian Federation

DSc (Medicine), professor, Head, Center for child neuropsychiatry and neurorehabilitation, neurologist, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation; N.F. Filatov Clinical Institute of Children’s Health, I.M. Sechenov First Moscow State Medical University (Sechenov University), Moscow, 119435, Russian Federation

Aleksey L. Kurenkov
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

DSc (Medicine), Head, Laboratory of nervous diseases, neurologist, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Eugeniya V. Uvakina
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

PhD (Medicine), Head, Department of neuropsychiatry and neurorehabilitation, neurologist, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Daria A. Fisenko
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

Neurologist, Center of child psychoneurology, National Medical Research Center of Children’s Health, Moscow, 119991, Russian Federation

e-mail: fisenko.daria@mail.ru

Vladislav V. Chernikov
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

PhD (Medicine), Head, Department of diagnostics and rehabilitation treatment, Head, Methodological accreditation and simulation center, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Elena L. Semikina
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

DSc (Medicine), Head, Laboratory department, chief researcher, doctor of laboratory diagnostics, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Sofia G. Popovich
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

Junior researcher, neurologist, Department of neuropsychiatry and neurorehabilitation, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Bella I. Bursagova
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

PhD (Medicine), neurologist, Department of neuropsychiatry and neurorehabilitation, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Oxana V. Globa
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

PhD (Medicine), senior researcher, neurologist, Department of neuropsychiatry and neurorehabilitation, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Natalya V. Andreenko
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

PhD (Medicine), neurologis, Department of neuropsychiatry and neurorehabilitation, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Luizat M. Abdullaeva
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

Junior researcher, neurologist, Department of neuropsychiatry and neurorehabilitation, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Julia A. Kurova
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

Neurologist, Department of neuropsychiatry and neurorehabilitation, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Nadezhda S. Adalimova
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

Neurologist, Department of neuropsychiatry and neurorehabilitation, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Daria S. Nikolenko
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

Neurologist, Department of neuropsychiatry and neurorehabilitation, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Anastasia A. Lyalina
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

Neurologist, Department of psychoneurology and neurorehabilitation of the National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

Olga V. Komarova
National Medical Research Center of Children’s Health of the Ministry, Health of the Russian Federation
Russian Federation

DSc (Medicine), nephrologist, First deputy director, Acting head, Institute of Medical Personnel Training, National Medical Research Center for Children’s Health, Moscow, 119991, Russian Federation

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