Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2...
Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age – a multicenter retrospective study
About this item
Full title
Author / Creator
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 and Syrbe, Steffen
Publisher
England: BioMed Central Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: BioMed Central Ltd
Subjects
More information
Scope and Contents
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...
Alternative Titles
Full title
Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age – a multicenter retrospective study
Authors, Artists and Contributors
Author / Creator
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
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_35616d559f1c44c9926976371af60411
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_35616d559f1c44c9926976371af60411
Other Identifiers
ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-019-1077-6
