Multicenter Phase 2 Trial of Sirolimus for Tuberous Sclerosis: Kidney Angiomyolipomas and Other Tumors Regress and VEGF- D Levels Decrease

BACKGROUND: Tuberous sclerosis (TSC) related tumors are characterized by constitutively activated mTOR signaling due to mutations in TSC1 or TSC2. METHODS: We completed a phase 2 multicenter trial to evaluate the efficacy and tolerability of the mTOR inhibitor, sirolimus, for the treatment of kidney...

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Detalles Bibliográficos
Autores principales: Dabora, Sandra L., Franz, David Neal, Ashwal, Stephen, Sagalowsky, Arthur, DiMario, Francis J., Miles, Daniel, Cutler, Drew, Krueger, Darcy, Uppot, Raul N., Rabenou, Rahmin, Camposano, Susana, Paolini, Jan, Fennessy, Fiona, Lee, Nancy, Woodrum, Chelsey, Manola, Judith, Garber, Judy, Thiele, Elizabeth A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2011
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3167813/
https://www.ncbi.nlm.nih.gov/pubmed/21915260
http://dx.doi.org/10.1371/journal.pone.0023379

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