Short-Term Safety and Efficacy of Onasemnogene Abeparvovec in 10 Patients with Spinal Muscular Atrophy: Cohort Study

Short-Term Safety and Efficacy of Onasemnogene Abeparvovec in 10 Patients with Spinal Muscular Atrophy: Cohort Study

Background. The efficacy and safety of onasemnogene abeparvovec have been demonstrated in patients with spinal muscular atrophy (SMA) in several clinical and observational studies. Gene replacement therapy results in Russian patients with SMA is not investigated yet.Objective. The aim of the study i...

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Bibliographic Details
Main Authors: Kristina S. Nevmerzhitskaya, Elena Yu. Sapego, Daria A. Morozova
Format: Article
Language:English
Published: "Paediatrician" Publishers LLC 2021-12-01
Series:Вопросы современной педиатрии
Subjects:
spinal muscular atrophy
children
onasemnogene abeparvovec
safety
liver transaminase
efficacy
сhop intend
Online Access:https://vsp.spr-journal.ru/jour/article/view/2807
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