Article
Inhibition of the hedgehog pathway in advanced basal-cell carcinoma.
The New England journal of medicine - 17 Sept 2009
Von Hoff Daniel D, LoRusso Patricia M, Rudin Charles M, Reddy Josina C, Yauch Robert L, Tibes Raoul, Weiss Glen J, Borad Mitesh J, Hann Christine L, Brahmer Julie R, Mackey Howard M, Lum Bertram L, Darbonne Walter C, Marsters James C, de Sauvage Frederic J, Low Jennifer A
Abstract excerpt
BACKGROUND: Mutations in hedgehog pathway genes, primarily genes encoding patched homologue 1 (PTCH1) and smoothened homologue (SMO), occur in basal-cell carcinoma. In a phase 1 clinical trial, we assessed the safety and pharmacokinetics of GDC-0449, a small-molecule inhibitor of SMO, and responses of metastatic or locally advanced basal-cell carcinoma to the drug. METHODS: We selected 33 patients with metastatic...
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