دورية أكاديمية

Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program

التفاصيل البيبلوغرافية
العنوان: Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program
المؤلفون: Giugliani, RobertoAff1, IDs1302302403176z_cor1, Gonzalez-Meneses, Antonio, Scarpa, Maurizio, Burton, Barbara, Wang, Raymond, Martins, Esmeralda, Oussoren, Esmeralda, Hennermann, Julia B., Chabrol, Brigitte, Grant, Christina L., Sun, Angela, Durand, Consuelo, Hetzer, Joel, Malkus, Betsy, Marsden, Deborah, Merritt II, J. Lawrence
المصدر: Orphanet Journal of Rare Diseases. 19(1)
قاعدة البيانات: Springer Nature Journals
الوصف
تدمد:17501172
DOI:10.1186/s13023-024-03176-z