PTC-124 (Ataluren) is an oxadiazole-based, orally available agent deisgned to selectively promote ribosomal readhtrough of premature stop codons, but not normal termination codons. [1] The minimal concentration of PTC124 showing observable readthrough was 0.01 - 0.1 uM (2.8 - 28 ng/ml), while maximal activity was observed at 3 uM (852 ng/ml). [2]

PTC-124 has been studied extensively in the treatment of cystic fibrosis and has shown good tolerability and efficacy in preclinical and clinical settings. [3, 4]