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...

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書誌詳細

出版年:	Нервно-мышечные болезни
主要な著者:	S. B. Artemyeva, Yu. O. Papina, O. A. Shidlovskaya, A. V. Monakhova, D. V. Vlodavets
フォーマット:	論文
言語:	ロシア語
出版事項:	ABV-press 2022-02-01
主題:	spinal muscular atrophy onasemnogene abeparvovec zolgensma® safety map program increased transaminases smn1 gene gene replacement therapy
オンライン･アクセス:	https://nmb.abvpress.ru/jour/article/view/474