Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age – a multicenter retrospective study-Reference-Cited by-同舟云学术

Safety and efficacy of mTOR inhibitor treatment in patients with tuberous sclerosis complex under 2 years of age – a multicenter retrospective study

Published:2019-05-03 Issue:1 Volume:14 Page:
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet J Rare Dis

Author:

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

Funder

Dietmar Hopp Stiftung

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics(clinical),General Medicine

Link

http://link.springer.com/content/pdf/10.1186/s13023-019-1077-6.pdf

Reference42 articles.

1. Crino PB, Nathanson KL, Henske EP. The tuberous sclerosis complex. N Engl J Med. 2006;355(13):1345–56.

2. Curatolo P, Moavero R, de Vries PJ. Neurological and neuropsychiatric aspects of tuberous sclerosis complex. Lancet Neurol. 2015;14(7):733–45.

3. Kingswood JC, d'Augeres GB, Belousova E, Ferreira JC, Carter T, Castellana R, et al. TuberOus SClerosis registry to increase disease awareness (TOSCA) - baseline data on 2093 patients. Orphanet J Rare Dis. 2017;12(1):2.