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Long-term safety and outcomes in hereditary tyrosinaemia type 1 with nitisinone treatment: a 15-year non-interventional, multicentre study.
Spiekerkoetter, Ute; Couce, Maria L; Das, Anibh M; de Laet, Corinne; Dionisi-Vici, Carlo; Lund, Allan M; Schiff, Manuel; Spada, Marco; Sparve, Erik; Szamosi, Johan; Vara, Roshni; Rudebeck, Mattias.
Afiliação
  • Spiekerkoetter U; Department of Paediatrics and Adolescent Medicine, University Medical Centre, Faculty of Medicine, University of Freiburg, Freiburg im Breisgau, Germany.
  • Couce ML; Hospital Clínico Universitario de Santiago de Compostela, Instituto de Investigación Sanitaria de Santiago de Compostela (IDIS), MetabERN, Santiago de Compostela, Spain.
  • Das AM; Department of Paediatrics, Hannover Medical School, Hannover, Germany.
  • de Laet C; Nutrition and Metabolism Unit, Department of Paediatrics, Hôpital Universitaire des Enfants Reine Fabiola, Brussels, Belgium.
  • Dionisi-Vici C; Division of Metabolism, Bambino Gesù Children's Hospital Instituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Rome, Italy.
  • Lund AM; Centre for Inherited Metabolic Diseases, Departments of Paediatrics and Clinical Genetics, Copenhagen University Hospital, Copenhagen, Denmark.
  • Schiff M; Necker Hospital, AP-HP, Reference Centre for Inborn Error of Metabolism (Filière G2M), Paediatrics Department, University of Paris, Paris, France; Inserm UMR_S1163, Institut Imagine, Paris, France.
  • Spada M; Department of Paediatrics, Regina Margherita Children Hospital, University of Torino, Torino, Italy.
  • Sparve E; Swedish Orphan Biovitrum (Sobi), Stockholm, Sweden.
  • Szamosi J; Swedish Orphan Biovitrum (Sobi), Stockholm, Sweden.
  • Vara R; Department of Paediatric Inherited Metabolic Disease, Evelina London Children's Hospital, London, UK.
  • Rudebeck M; Swedish Orphan Biovitrum (Sobi), Stockholm, Sweden. Electronic address: mattias.rudebeck@sobi.com.
Lancet Diabetes Endocrinol ; 9(7): 427-435, 2021 07.
Article em En | MEDLINE | ID: mdl-34023005
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Tirosinemias / Cicloexanonas / Inibidores Enzimáticos / Nitrobenzoatos Tipo de estudo: Clinical_trials / Diagnostic_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Tirosinemias / Cicloexanonas / Inibidores Enzimáticos / Nitrobenzoatos Tipo de estudo: Clinical_trials / Diagnostic_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Ano de publicação: 2021 Tipo de documento: Article