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| Verfasst von: | Saffari, Afshin [VerfasserIn]  |
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| | Brösse, Ines [VerfasserIn] |
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| | Tilburg, Cornelis M. van [VerfasserIn] |
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| | Hoffmann, Georg F. [VerfasserIn] |
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| | Kölker, Stefan [VerfasserIn] |
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| | Witt, Olaf [VerfasserIn] |
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| | Milde, Till [VerfasserIn] |
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| | Ziegler, Andreas [VerfasserIn] |
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| | Syrbe, Steffen [VerfasserIn] |
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| Titel: | 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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| Verf.angabe: | 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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| E-Jahr: | 2019 |
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| Jahr: | 03 May 2019 |
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| Umfang: | 13 S. |
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| Fussnoten: | Gesehen am 20.09.2019 |
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| Titel Quelle: | Enthalten in: Orphanet journal of rare diseases |
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| Ort Quelle: | London : BioMed Central, 2006 |
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| Jahr Quelle: | 2019 |
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| Band/Heft Quelle: | 14(2019), Artikel-ID 96, Seite 1-13 |
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| ISSN Quelle: | 1750-1172 |
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| Abstract: | 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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| DOI: | doi:10.1186/s13023-019-1077-6 |
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| URL: | kostenfrei: Volltext ; Verlag: https://doi.org/10.1186/s13023-019-1077-6 |
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| | kostenfrei: Volltext: https://doi.org/10.1186/s13023-019-1077-6 |
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| | DOI: https://doi.org/10.1186/s13023-019-1077-6 |
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| Datenträger: | Online-Ressource |
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| Sprache: | eng |
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| K10plus-PPN: | 167741944X |
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| Verknüpfungen: | → Zeitschrift |
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| Lokale URL UB: |  Zum Volltext |
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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 [VerfasserIn]; 03 May 2019 (Online-Ressource)
