A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States.
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| Title: | A retrospective cohort study describing the disease burden in patients with Pompe disease treated with enzyme replacement therapy in the United States. |
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| Authors: | Pillai NR; Division of Genetics & Metabolism, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA., Solomon F; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Steiner RD; Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, USA., Xie B; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Haselkorn T; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Young C; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Rozario N; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Walzer M; Astellas Pharma Global Development, Inc., Northbrook, IL, USA., Schoser B; Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University, Munich, Germany. |
| Source: | Journal of neuromuscular diseases [J Neuromuscul Dis] 2026 May; Vol. 13 (3), pp. 362-372. Date of Electronic Publication: 2025 Oct 28. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: IOS Press Country of Publication: United States NLM ID: 101649948 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2214-3602 (Electronic) Linking ISSN: 22143599 NLM ISO Abbreviation: J Neuromuscul Dis Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 2214-3602 |
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| DOI: | 10.1177/22143602251387210 |
