Figure 6.

SMO inhibitor predictions from mouse GEM models. A number of observations were made in GEM models that paralleled the experience with SMO inhibitors in the clinic. There was an initial dramatic elimination of large tumour mass. The tumour genotype accurately predicted the response to treatment. There was a rapid acquisition of resistance due to downstream activating mutations in the HH pathway. Developmental toxicities were observed in bone growth in young mice and young children.