20.500.12556/DiRROS-30030 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease a phase III open-label extension study (ATB200-07) glycogen storage disease type II alpha glucosidase myozyme n-butyldeoxynojirimycin Lysosomal storage disorders true false true Angleški jezik Ni določen Neznano 2026-06-12 09:47:36 2026-06-12 09:47:36 2026-06-13 03:52:11 0000-00-00 00:00:00 2024 0 0 Nasl. z nasl. zaslona; Opis z dne 31. 3. 2025; str. 2810-2823 Vol. 271 2024 0000-00-00 Zaloznikova Objavljeno NiDoloceno 0000-00-00 0000-00-00 0000-00-00 61 1432-1459 10.1007/s00415-024-12236-0 230906883 RAZ_Schoser_Benedikt_2024.pdf RAZ_Schoser_Benedikt_2024.pdf 1 861C1667179DF057CB6453B0A6F52111 8d7640a73bf1bd2dcae3ce271db4e8c540e27d853f46bc656fccb5bb6de05712 29fbaf92-6633-11f1-aebd-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=45283 https://link.springer.com/article/10.1007/s00415-024-12236-0 1 fc3fa94e-6632-11f1-aebd-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=45282 Univerzitetni klinični center Ljubljana 0 0 0