прогрессировать и, кажется, даже у носителей МДД. Серьезное, в конечном счете, летальное воздействие МДД из-за слабости сердечной, дыхательной мускулатуры и мышц конечностей обусловлено прогрессирующим фиброзом. Настоящие результаты показывают, что галофугинон имеет значительный потенциал, чтобы стать успешным фармакологическим подходом к сердечной и дыхательной дисфункции, наблюдаемой при МДД. Положительные результаты клинических испытаний галофугинона, как антифиброзного агента при МДД, по крайней мере, развернули возможности для эффективной терапии с использованием противовоспалительных и регенераторных препаратов, прекурсоров и стволовых клеток, и / или генетических конструкций. В заключение, лечение галофугиноном пожилых мышей MDX способствовало значительному структурному ремоделированию уменьшению фиброза, который в свою очередь поддерживает важные функциональные выгоды в мышцах конечностей, диафрагмы и сердца. Функциональный износ был ослаблен или обращен вспять, и экспрессия коллагена был сокращен. Это повышает устойчивость к физической нагрузке и улучшение восстановления мышц, дыхательных функциональных возможностей и сердечной функции. Эти результаты особенно важны, потому что препарат действовал одновременно на существующие ограничения на функции, связанные с фиброзом и снижение синтеза нового коллагена.
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