Лечение препаратом Вимизайм (Vimizim) подростка с синдромом Моркио А (мукополисахаридозом IV A)

Приведен клинический случай ферментозаместительной терапии препаратом элосульфаза альфа у подростка с редким лизосомным заболеванием – мукополисахаридозом типа IV A (синдромом МоркиоA). Лечение было начато довольно поздно, в возрасте12 лет на фоне тяжелого течения синдромa Моркио A. Темнеменее у ребенка отмечено улучшение показателей выносливости и мелкой моторики, повышение мышечного тонуса. Обсуждаются недостаточность информации о современных методах ферментозаместительной терапии, а также ограничения данного типа терапии. Подчеркивается необходимость междисциплинарного подхода для лечения таких заболеваний и облегчения состояния пациентов.

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