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 us...

Source: Orphanet Journal of Rare Diseases - May 3, 2019 Category: Internal Medicine Authors: Afshin Saffari, Ines Br össe, 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, And Tags: Research Source Type: research