Performance measures and patient/caregiver-reported assessments collected in a longitudinal, multi-center disease monitoring program (DMP) of patients with mucopolysaccharidosis type VII (MPS VII)
| Autor: | Malkus, Betsy C., Cohen, Jeanne M., Grant, Christina L., Scarpa, Maurizio, Sun, Angela, Wang, Raymond Y., Burton, Barbara K., Oussoren, Esmeralda, Martins, Esmeralda E., Durand, Consuelo, Chabrol, Brigitte, Ortiz, Lionel, Hetzer, Joel, Marsden, Deborah, Merritt, J. Lawrence, II, Giugliani, Roberto, Gonzalez-Meneses, Antonio |
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| Zdroj: | In Molecular Genetics and Metabolism February 2024 141(2) |
| Databáze: | ScienceDirect |
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