Based on promising early results showing a trend toward increased neurofibromin in primary NF1NS/+ Schwann cell cultures treated with gentamicin (Figures S3E and S3F), increased luciferase activity using a DLR (Figure 4A), and historical data supporting its use as a nonsense suppression therapy, we moved forward with evaluating the steady-state PK and PD of gentamicin in vivo in our minipig model of NF1. Here, NF1 is linked to neurofibromatosis type 1.