P261: Vestronidase alfa for the treatment of mucopolysaccharidosis VII (MPS VII): Updated results from a longitudinal, multicenter disease monitoring program (DMP)
| Autor: | Giugliani, Roberto, Gonzalez-Meneses, Antonio, Grant, Christina, Scarpa, Maurizio, Sun, Angela, Wang, Raymond, Burton, Barbara, van der Ploeg, Ans, Martins, Esmeralda, Durand, Consuelo, Chabrol, Brigitte, Hetzer, Joel, Marsden, Deborah, Merritt, J. Lawrence, II |
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| Zdroj: | In Genetics in Medicine Open 2024 2 Supplement 1 |
| Databáze: | ScienceDirect |
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