Disease characteristics, early effectiveness, and safety of vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII) assessed in a novel, longitudinal, multicenter Disease Monitoring Program (DMP)
| Autor: | Lau, Heather, Gonzalez-Meneses, Antonio, Scarpa, Maurizio, Hostutler, Robert, Zhang, Lin, Malkus, Betsy, Ramirez, Antonio Nino, Marsden, Deborah, Giugliani, Roberto |
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| Zdroj: | In Molecular Genetics and Metabolism February 2021 132(2):S63-S63 |
| Databáze: | ScienceDirect |
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