Abstract

The most frequent soft tissue cancer in children and adolescence is rhabdomyosarcoma. Alveolar RMS, which is driven by the fusion protein PAX3-FKHR or PAX7-FKHR, and embryonic RMS, which is frequently genetically diverse, are the two major histological subtypes of RMS. RMS prognosis has improved during the last several decades as a result of interdisciplinary care. However, therapy of patients with metastatic or resistant RMS has reached a plateau in recent years. Thus, improved understanding of the molecular and cellular biology of RMS, as well as the identification of novel treatment targets, are required to enhance the survival rate of RMS patients and their general well-being. In this review, we discuss the most recent advances in RMS molecular and cellular biology, such as changes in oncogenic pathways, miRNA, in vivo models, stem cells, and key signal transduction cascades implicated in the formation and progression of RMS. Furthermore, we highlight novel prospective targeted medicines that could improve RMS treatment