GDC-0449 (vismodegib), which acts by binding to the extracellular domain of SMO and antagonizing Hh signaling, has shown promising anti-tumor activity in advanced BCC and became the first Hh signaling inhibitor approved by the FDA in 2012 for the treatment of metastases and locally advanced BCC [15, 16]. The gene discussed is SMO; the disease is skin basal cell carcinoma.