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Pediatric Nephrology
Published in: Pediatric Nephrology 10/2015

01-10-2015 | Educational Review

Tuberous sclerosis complex: the past and the future

Authors: Liesbeth De Waele, Lieven Lagae, Djalila Mekahli

Published in: Pediatric Nephrology | Issue 10/2015

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Abstract

Renal lesions represent the second most significant cause of morbidity and mortality in patients with tuberous sclerosis complex (TSC). Recent advances in the understanding of the pathophysiology of TSC have led to the exploration of new potential therapeutic targets. Clinical trials with mammalian target of rapamycin (mTOR) inhibitors have demonstrated promising results for several indications, such as renal angiomyolipoma, subependymal giant cell astrocytoma, lymphangioleiomyomatosis and facial angiofibromas. Currently, there is a scarcity of natural history data and randomized, placebo-controlled clinical trials on TSC. Recently, however, recommendations for the diagnostic criteria, surveillance, and management of TSC patients have been updated. This review focuses on these novel recommendations and highlights the need for multidisciplinary follow-up of this multi-systemic disease.
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Metadata
Title
Tuberous sclerosis complex: the past and the future
Authors
Liesbeth De Waele
Lieven Lagae
Djalila Mekahli
Publication date
01-10-2015
Publisher
Springer Berlin Heidelberg
Published in
Pediatric Nephrology / Issue 10/2015
Print ISSN: 0931-041X
Electronic ISSN: 1432-198X
DOI
https://doi.org/10.1007/s00467-014-3027-9

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