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An Innovative Tool for Evidence-Based, Personalized Treatment Trials in Mucopolysaccharidosis.
Wiesinger, Anna-Maria; Bigger, Brian; Giugliani, Roberto; Lampe, Christina; Scarpa, Maurizio; Moser, Tobias; Kampmann, Christoph; Zimmermann, Georg; Lagler, Florian B.
Afiliação
  • Wiesinger AM; Institute of Congenital Metabolic Diseases, Paracelsus Medical University, 5020 Salzburg, Austria.
  • Bigger B; European Reference Network for Hereditary Metabolic Diseases, MetabERN, 33100 Udine, Italy.
  • Giugliani R; European Reference Network for Hereditary Metabolic Diseases, MetabERN, 33100 Udine, Italy.
  • Lampe C; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester M13 9PL, UK.
  • Scarpa M; Department of Genetics, Medical Genetics Service and Biodiscovery Laboratory, Portal Hospital de Clínicas de Porto Alegre, Universidade Federal do Rio Grande do Sul (UFRGS), Casa dos Raros, Porto Alegre 90610-261, Brazil.
  • Moser T; European Reference Network for Hereditary Metabolic Diseases, MetabERN, 33100 Udine, Italy.
  • Kampmann C; Department of Child Neurology, Epilepetology and Social Pediatrics, Center of Rare Diseases, University Hospital Giessen/Marburg, 35392 Giessen, Germany.
  • Zimmermann G; European Reference Network for Hereditary Metabolic Diseases, MetabERN, 33100 Udine, Italy.
  • Lagler FB; Regional Coordinating Center for Rare Diseases, University Hospital Udine, 33100 Udine, Italy.
Pharmaceutics ; 15(5)2023 May 22.
Article em En | MEDLINE | ID: mdl-37242808

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: Pharmaceutics Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Áustria

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: Pharmaceutics Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Áustria