Experience of using gene replacement therapy with Zolgensma® (onasemnogene abeparvovec) in real clinical practice in Russia

Experience of using gene replacement therapy with Zolgensma® (onasemnogene abeparvovec) in real clinical practice in Russia

Objective: to analyze the safety and evaluate the effectiveness of therapy with onasemnogene abeparvovec in patients with spinal muscular atrophy in real clinical practice based on the experience of using the drug in the neuromuscular center of Research Clinical Pediatric Institute of Pirogov Russia...

Full description

Bibliographic Details
Main Authors: S. B. Artemyeva, Yu. O. Papina, O. A. Shidlovskaya, A. V. Monakhova, D. V. Vlodavets
Format: Article
Language:Russian
Published: ABV-press 2022-02-01
Series:Нервно-мышечные болезни
Subjects:
spinal muscular atrophy
onasemnogene abeparvovec
zolgensma®
safety
map program
increased transaminases
smn1 gene
gene replacement therapy
Online Access:https://nmb.abvpress.ru/jour/article/view/474

Internet

https://nmb.abvpress.ru/jour/article/view/474

Similar Items