Rhabdomyosarcoma: development of molecular therapeutics under the microscope

Introduction Rhabdomyosarcoma (RMS), predominantly diagnosed in children, represents 3% of pediatric solid tumors. RMS has characteristics of skeletal muscle, although the cell of origin remains controversial. Cytotoxic therapeutics, radiation treatment and surgery remain the standard of care, however, outcomes for advanced disease have not changed for several decades. Major research advances over the past two decades have defined molecular subtypes and driver mutations that could provide new therapeutic targets.