Rhabdomyosarcoma (RMS) is the most frequent pediatric soft-tissue tumor accounting for
about 7% of childhood malignancies. Multimodal therapy is the standard treatment for individuals
with RMS but generally fails to cure high-risk group patients and can result in long-term side effects.
Therefore, understanding the mechanisms driving RMS might help to find new candidate targets for
more specific and effective therapeutic modalities. One of the molecular machineries which is often
deregulated in cancer and specifically involved in tumorigenesis of RMS, is Hedgehog (Hh) signaling.
There is increasing evidence that targeting this developmental pathway may hold promise in future
treatment strategies for RMS. In this review, we discuss the contribution of the Hh pathway in RMS, the challenges of inhibiting
this embryonic signaling in children with an update on recent preclinical data and ongoing clinical trials.
Keywords: Pediatric cancers, rhabdomyosarcoma, developmental pathways, hedgehog signaling, mouse models, targeted therapy.
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