104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe diseaseSchoser, Benedikt (Avtor) Kishnani, Priya S. (Avtor) Bratkovic, Drago (Avtor) Byrne, Barry J. (Avtor) Claeys, Kristl G. (Avtor) Diaz-Manera, Jordi (Avtor) Laforet, Pascal (Avtor) Roberts, Mark (Avtor) Toscano, Antonio (Avtor) Van der Ploeg, Ans. T. (Avtor) Koritnik, Blaž (Sodelavec pri raziskavi) glycogen storage disease type IIalpha glucosidasemyozymen-butyldeoxynojirimycinLysosomal storage disorders20242026-06-12 09:47:36Neznano30030UDK: 61ISSN pri članku: 1432-1459DOI: 10.1007/s00415-024-12236-0COBISS_ID: 230906883sl