CFTR and neuroblastoma: Therefore, in this study, we investigated if the off-label mechanism of Vx-809 is also confirmed in a neuroblastoma cell model, often used as in vitro models of neuronal function and differentiation, leading to the hypothesis that the drug interacts not only with the CFTR protein but, by an unknown mechanism, also on other misfolded proteins, demonstrating that it can also be used in other diseases, like NDs (Figure 1).