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Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study.
Saffari, Afshin; Brösse, Ines; Wiemer-Kruel, Adelheid; Wilken, Bernd; Kreuzaler, Paula; Hahn, Andreas; Bernhard, Matthias K; van Tilburg, Cornelis M; Hoffmann, Georg F; Gorenflo, Matthias; Hethey, Sven; Kaiser, Olaf; Kölker, Stefan; Wagner, Robert; Witt, Olaf; Merkenschlager, Andreas; Möckel, Andreas; Roser, Timo; Schlump, Jan-Ulrich; Serfling, Antje; Spiegler, Juliane; Milde, Till; Ziegler, Andreas; Syrbe, Steffen.
Afiliação
  • Saffari A; Division of Child Neurology and Metabolic Medicine, Center for Paediatrics and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.
  • Brösse I; Division of Child Neurology and Metabolic Medicine, Center for Paediatrics and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.
  • Wiemer-Kruel A; Clinic for Children and Adolescents, Epilepsy Centre Kork, Kehl-Kork, Germany.
  • Wilken B; Department of Pediatric Neurology, Kassel Hospital, Kassel, Germany.
  • Kreuzaler P; Department of Child Neurology, University Hospital, Gießen, Germany.
  • Hahn A; Department of Child Neurology, University Hospital, Gießen, Germany.
  • Bernhard MK; Department of Neuropediatrics, University Hospital of Children, Leipzig, Germany.
  • van Tilburg CM; Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Hoffmann GF; Clinical Cooperation Unit Pediatric Oncology, German Cancer Research Center (DKFZ) and German Consortium for Translational Cancer Research (DKTK), Heidelberg, Germany.
  • Gorenflo M; KiTZ Clinical Trial Unit (ZIPO), Department of Pediatric Hematology and Oncology, Heidelberg University Hospital, Heidelberg, Germany.
  • Hethey S; Division of Child Neurology and Metabolic Medicine, Center for Paediatrics and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.
  • Kaiser O; Department for Congenital Heart Defects/Paediatric Cardiology, Heidelberg University Hospital, Heidelberg, Germany.
  • Kölker S; Auf der Bult - Center for Children and Adolescents, Hannover, Germany.
  • Wagner R; Department of Paediatrics I, Paediatric Neurology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany.
  • Witt O; Division of Child Neurology and Metabolic Medicine, Center for Paediatrics and Adolescent Medicine, University Hospital Heidelberg, Heidelberg, Germany.
  • Merkenschlager A; Department of Pediatric Cardiology, University of Leipzig, Heart Center, Leipzig, Germany.
  • Möckel A; Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
  • Roser T; Clinical Cooperation Unit Pediatric Oncology, German Cancer Research Center (DKFZ) and German Consortium for Translational Cancer Research (DKTK), Heidelberg, Germany.
  • Schlump JU; KiTZ Clinical Trial Unit (ZIPO), Department of Pediatric Hematology and Oncology, Heidelberg University Hospital, Heidelberg, Germany.
  • Serfling A; Department of Neuropediatrics, University Hospital of Children, Leipzig, Germany.
  • Spiegler J; Sana Kliniken Leipziger Land, Borna, Germany.
  • Milde T; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, University of Munich, Munich, Germany.
  • Ziegler A; Division for Children and Adolescents, Evangelical Hospital Oberhausen, Oberhausen, Germany.
  • Syrbe S; Sana Kliniken Leipziger Land, Borna, Germany.
Orphanet J Rare Dis ; 14(1): 96, 2019 05 03.
Article em En | MEDLINE | ID: mdl-31053163

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Esclerose Tuberosa / Serina-Treonina Quinases TOR Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Alemanha País de publicação: Reino Unido

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Esclerose Tuberosa / Serina-Treonina Quinases TOR Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Child / Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Alemanha País de publicação: Reino Unido