Катрен Стиль
В России зарегистрирован первый препарат для лечения спинальной мышечной атрофии
Лекарственное средство «Спинраза» (МНН: нусинерсен), предназначенное для терапии спинальной мышечной атрофии (СМА), получило регистрацию в России. Соответствующая информация опубликована 16 августа в Государственном реестре лекарственных средств.
Нусинерсен является антисмысловым олигонуклеотидом, структура которого специально разработана для лечения СМА. В основе заболевания лежит мутация, которая приводит к утрате функции гена SMN1, отвечающего за выживание двигательных нейронов, из‑за чего в организме происходит дефицит белка SMN. За продукцию этого белка также «отвечает» ген SMN2, но он вырабатывает его в меньшем количестве. Недостаток белка приводит к нарушениям работы мускулатуры ног, головы и шеи.
Нусинерсен связывается с геном SMN2, вытесняя факторы сплайсинга, которые в норме подавляют его. Таким образом, ЛС фактически «переделывает» ген SMN2 в SMN1 по функциональной части. В итоге повышается содержание белка SMN в центральной нервной системе.
Нусинерсен является первым в мире препаратом, созданным для лечения спинальной мышечной атрофии. Впервые он был одобрен к применению в 2016 году в США. Регистрация лекарства проходила в ускоренном режиме, поскольку препарат имеет статус орфанного ЛС.
Также ранее в США был одобрен препарат «Zolgensma», предназначенный специально для лечения СМА у детей в возрасте до двух лет.
21 августа 2019
Фото: depositphotos.com
Выпуск: №187, август 20193742 просмотров
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