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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
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| Veröffentlicht in: | Orphanet journal of rare diseases 14(2019), Artikel-ID 96, Seite 1-13 |
|---|---|
| Personen und Körperschaften: | , , , , , , , , |
| Titel: | Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study/ Afshin Saffari, Ines Brösse, Adelheid Wiemer-Kruel, Bernd Wilken, Paula Kreuzaler, Andreas Hahn, Matthias K. Bernhard, Cornelis M. van Tilburg, Georg F. Hoffmann, Matthias Gorenflo, Sven Hethey, Olaf Kaiser, Stefan Kölker, Robert Wagner, Olaf Witt, Andreas Merkenschlager, Andreas Möckel, Timo Roser, Jan-Ulrich Schlump, Antje Serfling, Juliane Spiegler, Till Milde, Andreas Ziegler and Steffen Syrbe |
| Format: | E-Book-Kapitel |
| Sprache: | Englisch |
| veröffentlicht: |
03 May 2019
|
| Gesamtaufnahme: |
: Orphanet journal of rare diseases, 14(2019), Artikel-ID 96, Seite 1-13
, volume:14 |
| Quelle: | Verbunddaten SWB Lizenzfreie Online-Ressourcen |
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| 245 | 1 | 0 | |a Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study |c Afshin Saffari, Ines Brösse, Adelheid Wiemer-Kruel, Bernd Wilken, Paula Kreuzaler, Andreas Hahn, Matthias K. Bernhard, Cornelis M. van Tilburg, Georg F. Hoffmann, Matthias Gorenflo, Sven Hethey, Olaf Kaiser, Stefan Kölker, Robert Wagner, Olaf Witt, Andreas Merkenschlager, Andreas Möckel, Timo Roser, Jan-Ulrich Schlump, Antje Serfling, Juliane Spiegler, Till Milde, Andreas Ziegler and Steffen Syrbe |
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| 520 | |a Tuberous sclerosis complex (TSC) is a multisystem disease with prominent neurologic manifestations such as epilepsy, cognitive impairment and autism spectrum disorder. mTOR inhibitors have successfully been used to treat TSC-related manifestations in older children and adults. However, data on their safety and efficacy in infants and young children are scarce. The objective of this study is to assess the utility and safety of mTOR inhibitor treatment in TSC patients under the age of 2 years. | ||
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| contents | Tuberous sclerosis complex (TSC) is a multisystem disease with prominent neurologic manifestations such as epilepsy, cognitive impairment and autism spectrum disorder. mTOR inhibitors have successfully been used to treat TSC-related manifestations in older children and adults. However, data on their safety and efficacy in infants and young children are scarce. The objective of this study is to assess the utility and safety of mTOR inhibitor treatment in TSC patients under the age of 2 years. |
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| spelling | Saffari, Afshin 1988- VerfasserIn (DE-588)1076753302 (DE-627)835291251 (DE-576)445631406 aut, Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study Afshin Saffari, Ines Brösse, Adelheid Wiemer-Kruel, Bernd Wilken, Paula Kreuzaler, Andreas Hahn, Matthias K. Bernhard, Cornelis M. van Tilburg, Georg F. Hoffmann, Matthias Gorenflo, Sven Hethey, Olaf Kaiser, Stefan Kölker, Robert Wagner, Olaf Witt, Andreas Merkenschlager, Andreas Möckel, Timo Roser, Jan-Ulrich Schlump, Antje Serfling, Juliane Spiegler, Till Milde, Andreas Ziegler and Steffen Syrbe, 03 May 2019, 13, Text txt rdacontent, Computermedien c rdamedia, Online-Ressource cr rdacarrier, Gesehen am 20.09.2019, Tuberous sclerosis complex (TSC) is a multisystem disease with prominent neurologic manifestations such as epilepsy, cognitive impairment and autism spectrum disorder. mTOR inhibitors have successfully been used to treat TSC-related manifestations in older children and adults. However, data on their safety and efficacy in infants and young children are scarce. The objective of this study is to assess the utility and safety of mTOR inhibitor treatment in TSC patients under the age of 2 years., Brösse, Ines VerfasserIn (DE-588)1191494470 (DE-627)1670114147 aut, Tilburg, Cornelis M. van VerfasserIn (DE-588)1144664527 (DE-627)1004962355 (DE-576)495479519 aut, Hoffmann, Georg F. 1957- VerfasserIn (DE-588)115652868 (DE-627)077386116 (DE-576)261230042 aut, Kölker, Stefan VerfasserIn (DE-588)1022937758 (DE-627)717335771 (DE-576)366197568 aut, Witt, Olaf 1965- VerfasserIn (DE-588)17275030X (DE-627)697681750 (DE-576)133607410 aut, Milde, Till VerfasserIn (DE-588)1038311837 (DE-627)75711234X (DE-576)39229334X aut, Ziegler, Andreas 1979- VerfasserIn (DE-588)134123530 (DE-627)560894201 (DE-576)278444253 aut, Syrbe, Steffen 1976- VerfasserIn (DE-588)133581926 (DE-627)691603138 (DE-576)272999482 aut, Enthalten in Orphanet journal of rare diseases London : BioMed Central, 2006 14(2019), Artikel-ID 96, Seite 1-13 Online-Ressource (DE-627)50900637X (DE-600)2225857-7 (DE-576)260614424 1750-1172 nnns, volume:14 year:2019 elocationid:96 pages:1-13 extent:13, https://doi.org/10.1186/s13023-019-1077-6 Verlag Resolving-System kostenfrei Volltext, https://doi.org/10.1186/s13023-019-1077-6 Verlag kostenfrei Volltext, https://doi.org/10.1186/s13023-019-1077-6 LFER, LFER 2019-10-07T00:00:00Z |
| spellingShingle | Saffari, Afshin, Brösse, Ines, Tilburg, Cornelis M. van, Hoffmann, Georg F., Kölker, Stefan, Witt, Olaf, Milde, Till, Ziegler, Andreas, Syrbe, Steffen, Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study, Tuberous sclerosis complex (TSC) is a multisystem disease with prominent neurologic manifestations such as epilepsy, cognitive impairment and autism spectrum disorder. mTOR inhibitors have successfully been used to treat TSC-related manifestations in older children and adults. However, data on their safety and efficacy in infants and young children are scarce. The objective of this study is to assess the utility and safety of mTOR inhibitor treatment in TSC patients under the age of 2 years. |
| title | Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study |
| title_auth | Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study |
| title_full | Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study Afshin Saffari, Ines Brösse, Adelheid Wiemer-Kruel, Bernd Wilken, Paula Kreuzaler, Andreas Hahn, Matthias K. Bernhard, Cornelis M. van Tilburg, Georg F. Hoffmann, Matthias Gorenflo, Sven Hethey, Olaf Kaiser, Stefan Kölker, Robert Wagner, Olaf Witt, Andreas Merkenschlager, Andreas Möckel, Timo Roser, Jan-Ulrich Schlump, Antje Serfling, Juliane Spiegler, Till Milde, Andreas Ziegler and Steffen Syrbe |
| title_fullStr | Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study Afshin Saffari, Ines Brösse, Adelheid Wiemer-Kruel, Bernd Wilken, Paula Kreuzaler, Andreas Hahn, Matthias K. Bernhard, Cornelis M. van Tilburg, Georg F. Hoffmann, Matthias Gorenflo, Sven Hethey, Olaf Kaiser, Stefan Kölker, Robert Wagner, Olaf Witt, Andreas Merkenschlager, Andreas Möckel, Timo Roser, Jan-Ulrich Schlump, Antje Serfling, Juliane Spiegler, Till Milde, Andreas Ziegler and Steffen Syrbe |
| title_full_unstemmed | Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study Afshin Saffari, Ines Brösse, Adelheid Wiemer-Kruel, Bernd Wilken, Paula Kreuzaler, Andreas Hahn, Matthias K. Bernhard, Cornelis M. van Tilburg, Georg F. Hoffmann, Matthias Gorenflo, Sven Hethey, Olaf Kaiser, Stefan Kölker, Robert Wagner, Olaf Witt, Andreas Merkenschlager, Andreas Möckel, Timo Roser, Jan-Ulrich Schlump, Antje Serfling, Juliane Spiegler, Till Milde, Andreas Ziegler and Steffen Syrbe |
| title_in_hierarchy | Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study / Afshin Saffari, Ines Brösse, Adelheid Wiemer-Kruel, Bernd Wilken, Paula Kreuzaler, Andreas Hahn, Matthias K. Bernhard, Cornelis M. van Tilburg, Georg F. Hoffmann, Matthias Gorenflo, Sven Hethey, Olaf Kaiser, Stefan Kölker, Robert Wagner, Olaf Witt, Andreas Merkenschlager, Andreas Möckel, Timo Roser, Jan-Ulrich Schlump, Antje Serfling, Juliane Spiegler, Till Milde, Andreas Ziegler and Steffen Syrbe, |
| title_short | Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age - a multicenter retrospective study |
| title_sort | safety and efficacy of mtor inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age a multicenter retrospective study |
| url | https://doi.org/10.1186/s13023-019-1077-6 |