VEGFA and infantile convulsions and choreoathetosis: VEGF/VEGFR-targeted therapies have not demonstrated clinically meaningful efficacy as monotherapy and are not currently standard of care for this indication: early-phase trials of VEGF pathway inhibitors, such as sorafenib, sunitinib, and regorafenib, have shown limited activity in iCCA, with low objective response rates and modest progression-free survival, and are not recommended as single agents [62,63,64].