| Title: | 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) |
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| Authors: | ID Schoser, Benedikt (Author)
ID Kishnani, Priya S. (Author)
ID Bratkovic, Drago (Author)
ID Byrne, Barry J. (Author)
ID Claeys, Kristl G. (Author)
ID Diaz-Manera, Jordi (Author)
ID Laforet, Pascal (Author)
ID Roberts, Mark (Author)
ID Toscano, Antonio (Author)
ID Van der Ploeg, Ans. T. (Author)
ID Koritnik, Blaž (Research coworker), et al. |
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| Files: |  PDF - Presentation file, download (922,82 KB)
MD5: 861C1667179DF057CB6453B0A6F52111
 URL - Source URL, visit https://link.springer.com/article/10.1007/s00415-024-12236-0
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| Language: | English |
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| Typology: | 1.01 - Original Scientific Article |
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| Organization: |  UKC LJ - Ljubljana University Medical Centre
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| Keywords: | glycogen storage disease type II, alpha glucosidase, myozyme, n-butyldeoxynojirimycin, Lysosomal storage disorders |
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| Publication status: | Published |
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| Publication version: | Version of Record |
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| Year of publishing: | 2024 |
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| Number of pages: | str. 2810-2823 |
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| Numbering: | Vol. 271 |
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| PID: | 20.500.12556/DiRROS-30030  |
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| UDC: | 61 |
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| ISSN on article: | 1432-1459 |
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| DOI: | 10.1007/s00415-024-12236-0 |
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| COBISS.SI-ID: | 230906883 |
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| Note: | Nasl. z nasl. zaslona;
Opis z dne 31. 3. 2025;
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| Publication date in DiRROS: | 12.06.2026 |
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| Views: | 78 |
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| Downloads: | 74 |
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| Metadata: |    |
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