Onasemnogene abeparvovec gene replacement therapy for spinal muscular atrophy in children: a Moscow experience
This study presents the findings of a long-term assessment of the efficacy and safety profile of drug OA in pediatric patients across various age groups and functional ability levels, including individuals identified through expanded newborn screening. Younger patients exhibited a significantly lower incidence of adverse events and demonstrated superior therapeutic efficacy. The majority of older children experienced either positive progression or stabilization of their condition, outcomes that were largely contingent upon their initial functional status and the duration of the pre-treatment waiting period.
Authors
- Vera Zarubina (ORCID: https://orcid.org/0000-0002-9724-7653)
- Н. Л. Печатникова (ORCID: https://orcid.org/0000-0002-2445-132X)
- В.В. Горев (ORCID: https://orcid.org/0000-0001-8272-3648)
- A. A. Rakhalina (ORCID: https://orcid.org/0000-0002-3263-5145)
- С И Валиева (ORCID: https://orcid.org/0009-0009-6241-9142)
- N. A. POLYAKOVA (ORCID: https://orcid.org/0000-0003-2255-6096)
- В. С. Какаулина (ORCID: https://orcid.org/0000-0002-3764-2766)
- Tatiana N. Kekeeva (ORCID: https://orcid.org/0000-0002-4986-5748)
- Yu. E. Martynenko (ORCID: https://orcid.org/0000-0002-0899-9627)
- Анна Владимировна Власова (ORCID: https://orcid.org/0000-0001-5272-2070)
- I.P. Vitkovskaya (ORCID: https://orcid.org/0000-0002-0740-1558)
- Z.I. Esmurzieva (ORCID: https://orcid.org/0009-0004-4806-4545)
- A.A. Chudaykina (ORCID: https://orcid.org/0009-0003-1546-9258)
Institutions
- Pirogov Russian National Research Medical University (RU)
- Central Research Institute (IN)
- Federal Research Institute for Health Organization and Informatics (RU)
- Research Centre for Medical Genetics (RU)
- City Clinical Hospital (RU)
Publication Details
- Journal
- S S Korsakov Journal of Neurology and Psychiatry
- Published
- 2025-11-21
- DOI
- https://doi.org/10.17116/jnevro2025125102103
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00