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)

التفاصيل البيبلوغرافية
العنوان:	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)
المؤلفون:	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
المصدر:	In Molecular Genetics and Metabolism February 2024 141(2)
قاعدة البيانات:	ScienceDirect

الوصف
تدمد:	10967192
DOI:	10.1016/j.ymgme.2023.107949