[3] When tested in the nonsense mutation mdx mouse model of DMD, ataluren generated production of full-length, functional dystrophin protein. [3], [4] We and others have shown that ataluren is active in multiple cell-based and animal disease models.[3]–[9] Phase 1 studies in healthy volunteers established the initial ataluren safety profile [10] and defined dosing regimens for achieving target trough plasma concentrations (2 to 10 μg/mL) known to be active in preclinical models [3], [5]. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.