На WMS 2026 представлены пятилетние результаты ферментозаместительной терапии болезни Помпе комбинацией cipaglucosidase alfa и miglustat, свидетельствующие об относительной стабилизации двигательной и дыхательной функций https://investors.biomarin.com/news/news-details/2026/BioMarin-Announces-Five-Year-Phase-3-Data-for-POMBILITI-cipaglucosidase-alfa-atga--OPFOLDA-miglustat-at-World-Muscle-Society-Annual-Congress/default.aspx
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