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Profile: Linearized PDF PDFMetadata: Objects: 437 FreeObjects: 1 IncrementalUpdates: 1 DocumentCatalog: ViewerPreferences: HideToolbar: false HideMenubar: false HideWindowUI: false FitWindow: false CenterWindow: false DisplayDocTitle: false NonFullScreenPageMode: UseNone Direction: L2R ViewArea: CropBox ViewClip: CropBox PrintArea: CropBox PageClip: CropBox PageLayout: SinglePage PageMode: UseOutlines Language: EN Outlines: Item: Title: STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosi搀愀猀攀 愀氀昀愩㸾൥湤潢樍㌷㔠〠潢樍㰼⽄⠴ㄵ弲〲ㅟ㄰㐰㥟䅲瑩捬攮楮摤㩁湣桯爠ㄺ㜴 Children: Item: Title: Abstract Children: Item: Title: Background Item: Title: Methods Item: Title: Findings Item: Title: Conclusions Item: Title: Background Item: Title: Methods Children: Item: Title: Study centers and inclusion criteria Item: Title: Study procedures and clinical assessments Children: Item: Title: Muscle strength Item: Title: 6-min walk test Item: Title: Pulmonary function assessments Item: Title: Individual level Item: Title: Safety Item: Title: Statistical analysis Item: Title: Results Children: Item: Title: Patients and baseline characteristics Item: Title: Muscle strength Item: Title: 6-min walk test Item: Title: Lung function assessments Item: Title: Individual level Item: Title: Long-term data longer than 10 years under ERT Item: Title: Supportive devices Children: Item: Title: Ventilation status Item: Title: Wheelchair use Item: Title: Long-term side effects Item: Title: Discussion Item: Title: Conclusion Item: Title: Acknowledgements Item: Title: References Info: Title: STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa Author: Kristina Gutschmidt Subject: Journal of Neurology, https://doi.org/10.1007/s00415-021-10409-9 Keywords: Alglucosidase alpha,Efficacy,Enzyme replacement therapy,Glycogen storage disease type 2,Long term follow-up,Pompe disease 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long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa Kristina Gutschmidt Olimpia Musumeci Jordi Díaz-Manera Yin-Hsiu Chien Karl Christian Knop Stephan Wenninger Federica Montagnese Alessia Pugliese Graziana Tavilla Jorge Alonso-Pérez Paul Wuh-Liang Hwu Antonio Toscano Benedikt Schoser 10.1007/s00415-021-10409-9 2010-04-23 true springer.com springerlink.com https://doi.org/10.1007/s00415-021-10409-9 10.1007/s00415-021-10409-9 1432-1459 268 7 2482 2492 journal Journal of Neurology The Author(s) 2010-04-23 true 10.1007/s00415-021-10409-9 noindex springer.com springerlink.com VoR uuid:85f9275e-6e24-432e-80fd-24712b445bf0 uuid:b258eee1-11d0-454c-a028-a16ec9886277 default 1 converted uuid:54669e73-3a9d-4d2a-8753-74b3d009b669 converted to PDF/A-2b pdfToolbox 2021-06-16T17:43:13+05:30 converted uuid:5655a21e-1468-4e60-86eb-f234da2b4bed converted to PDF/A-2b pdfToolbox 2021-06-16T17:43:45+05:30 2 B Benedikt Schoser 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