Small-molecule inhibitors of TGFBR, pSMAD or BMP signaling could prevent EndMT and reduce the size and number of cerebral malformations, demonstrating that dysregulation of TGF-β/BMP signaling directly contributes to the onset and pathological process of CCMs (Maddaluno et al., 2013). This evidence concerns the gene TGFB1 and cerebrocostomandibular syndrome.