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Abstract
A majority of individuals with BCNS have germline mutations affecting PTCH1, and BCCs that develop in these individuals are highly responsive to Smoothened inhibitors. However, BCCs which develop in minority of BCNS patients with underlying SUFU mutations may be less responsive to Smoothened inhibitors because inactivation of SUFU is downstream of SMO. Development of next-generation HH antagonists that target components downstream of SMO are under development for efficacy in Smoothened inhibitor resistant BCCs and may have efficacy in BCCs with SUFU mutations.
View details for DOI 10.1016/j.jid.2019.06.139
View details for PubMedID 31330148