Among SMO inhibitors, GDC-0449 and LDE-225 are in the clinic for the treatment of advanced BCC with aberrant HH activity due to loss of the functional allele of PTCH [297,298] The steroidal alkaloid, cyclopamine, has progressively shown therapeutic potential as an inhibitor for HH signaling. The gene discussed is PTCH1; the disease is skin basal cell carcinoma.