New Treatments for Rhabdomyosarcoma: the Importance of Target Practice

To address the poor outcomes in rhabdomyosarcoma, particularly the alveolar subtype, new therapies are needed. Potential cancer-specific alterations that may be molecular targets include gene fusions or copy number changes. Following the latter strategy, an attractive antigene approach was developed to inhibit MYCN oncogene expression in rhabdomyosarcoma.