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BIBTEX RIS

Inhibition of the Hedgehog Pathway in Advanced Basal-Cell Carcinoma

2009; Massachusetts Medical Society; Volume: 361; Issue: 12 Linguagem: Inglês

10.1056/nejmoa0905360

ISSN

1533-4406

Autores

Daniel D. Von Hoff, Patricia LoRusso, Charles M. Rudin, Josina C. Reddy, Robert L. Yauch, Raoul Tibes, Glen J. Weiss, Mitesh J. Borad, Christine L. Hann, Julie R. Brahmer, Howard Mackey, B. Lum, Walter C. Darbonne, James C. Marsters, Frédéric J. de Sauvage, Jennifer A. Low,

Tópico(s)

Genetic and rare skin diseases.

Resumo

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.

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