In recent years, advancements in the understanding of the molecular pathways of chordomas have paved the way for the adoption of several targeted therapies, focusing on receptor tyrosine kinases such as epidermal growth factor receptor (EGFR), platelet-derived growth factor receptor (PDGFR), and brachyury, a transcription factor almost exclusively expressed in chordomas [6]. The gene discussed is TBX1; the disease is chordoma.