Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL
| Autor: | Mozaffar, Tahseen, Bratkovic, Drago, Byrne, Barry J., Claeys, Kristl G., Diaz-Manera, Jordi, Dimachkie, Mazen M., Kishnani, Priya S., Kushlaf, Hani, Roberts, Mark E., Toscano, Antonio, Castelli, Jeffrey, Holdbrook, Fred K., Das, Sheela Sitaraman, Wasfi, Yasmine, Schoser, Benedikt |
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| Zdroj: | In Molecular Genetics and Metabolism February 2024 141(2) |
| Databáze: | ScienceDirect |
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