Regarding the efficacy of Hh inhibitors, an in vivo study was carried out to assess the effectiveness of sonidegib (Smo inhibitor) treatment in the embryonal subtype of rhabdomyosarcoma (ERMS) with a mutation in Ptch. Sonidegib had a significant antitumor effect in murine models of ERMS, as evidenced by a reduction of tumor growth in monotherapy and combined with pictilisib, a PI3K inhibitor. This evidence concerns the gene SMO and neoplasm.