Figure 4. PTC299 inhibits DHODH activity and reduces elevated VEGFA levels in patients with neurofibromatosis type 2.
(A) Reduction in mean levels of serum VEGFA after 8 weeks of PTC299 treatment; Data are represented as mean ± SEM, n = 11. (B) VEGFA reduction for individual patients over the course of the PTC299 treatment; (C) Serum levels of DHO are correlated with PTC299 concentration in patient-002; (D) Serum levels of DHO are inversely correlated with serum VEGFA levels in patient-002. (E) PTC299 increases serum DHO in three additional patients whose serum samples were still available at the time of analysis. BLQ: below the level of quantification.