ppharmПедиатрическая фармакологияPediatric pharmacology1727-57762500-3089Издательство «ПедиатрЪ»10.15690/pf.v11i6.1215ppharm-35Research ArticleОБЗОР ЛИТЕРАТУРЫREVIEWЭФФЕКТИВНОСТЬ И БЕЗОПАСНОСТЬ ПРИМЕНЕНИЯ ВЕЛАГЛЮЦЕРАЗЫ АЛЬФА У ПАЦИЕНТОВ С БОЛЕЗНЬЮ ГОШЕ 1-ГО ТИПА (ПО ДАННЫМ МЕЖДУНАРОДНЫХ ИССЛЕДОВАНИЙ)EFFECTIVENESS AND SAFETY OF VELAGLUCERASE ALFA IN TREATMENT OF GAUCHER DISEASE TYPE 1 (ACCORDING TO INTERNATIONAL STUDIES)ГундобинаО. С.GundobinaO. S.

кандидат медицинских наук, заведующая отделением восстановительного лечения детей с болезнями органов пищеварительной системы НИИ профилактической педиатрии и восстановительного лечения ФГБНУ «Научный центр здоровья детей»Адрес: 119991, Москва, Ломоносовcкий проспект, д. 2, тел.: +7 (499) 134-01-57

gundobina@nczd.ruМовсисянГ. Б.MovsisyanG. B.gundobina@nczd.ruКомароваЕ. В.KomarovaE. V.gundobina@nczd.ruНамазова-БарановаЛ. С.Namazova-BaranovaL. S.namazova@nczd.ruНаучный центр здоровья детей, Москва, Российская ФедерацияРоссияScientific Center of Children’s Health, Moscow, Russian FederationRussian Federation201419112014116Педиатрическая фармакология5255Copyright © Гундобина О.С., Мовсисян Г.Б., Комарова Е.В., Намазова-Баранова Л.С., 20142014Гундобина О.С., Мовсисян Г.Б., Комарова Е.В., Намазова-Баранова Л.С.Gundobina O.S., Movsisyan G.B., Komarova E.V., Namazova-Baranova L.S.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.pedpharma.ru/jour/article/view/35

Статья посвящена современным подходам к лечению болезни Гоше. Авторами отражены основные аспекты заболевания, представлены данные по истории создания и внедрения в клиническую практику патогенетической ферментозаместительной терапии. Обзор международных клинических исследований демонстрирует высокую эффективность и безопасность длительной ферментозаместительной терапии препаратом велаглюцераза альфа у пациентов с подтвержденным диагнозом болезнь Гоше 1-го типа.

 

The article is dedicated to modern approaches to treatment of Gaucher’s disease. The authors list the primary aspects of the disease and present data on the origin and introduction of pathogenetic enzyme replacement therapy to clinical practice. Review of international clinical studies demonstrates high effectiveness and safety of long-term enzyme replacement therapy with velaglucerase alfa in patients with confirmed type I Gaucher’s disease.

 

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The authors declare that there are no conflicts of interest present.