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Miglustat in patients with Niemann-Pick disease Type C (NP-C): a multicenter observational retrospective cohort study.
Pineda, M; Wraith, J E; Mengel, E; Sedel, F; Hwu, W-L; Rohrbach, M; Bembi, B; Walterfang, M; Korenke, G C; Marquardt, T; Luzy, C; Giorgino, R; Patterson, M C.
Affiliation
  • Pineda M; Hospital Sant Joan de Deu, Barcelona, Spain. pineda@hsjdbcn.org
Mol Genet Metab ; 98(3): 243-9, 2009 Nov.
Article in En | MEDLINE | ID: mdl-19656703
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: 1-Deoxynojirimycin / Enzyme Inhibitors / Niemann-Pick Disease, Type C Type of study: Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Limits: Adolescent / Child / Female / Humans / Male Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2009 Document type: Article Affiliation country: España Country of publication: Estados Unidos
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: 1-Deoxynojirimycin / Enzyme Inhibitors / Niemann-Pick Disease, Type C Type of study: Etiology_studies / Incidence_studies / Observational_studies / Risk_factors_studies Limits: Adolescent / Child / Female / Humans / Male Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2009 Document type: Article Affiliation country: España Country of publication: Estados Unidos