2​ Biological Activity PTC124 (Ataluren) targets genetic disorders caused by nonsense mutations. [1] PTC124 is an orally bioavailable compound that promotes of the human CFTR-G542X nonsense allele in a CF mouse model. [2] Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial. [3] PTC124 induces dose-dependent suppression of premature translational termination without concomitant effects on normal termination or mRNA decay. [1]In safety pharmacology studies in rats and dogs, oral administration of PTC124 induces no adverse neurological, pulmonary, or cardiovascular effects at doses through 1500 mg/kg. In toxicology studies in rats and dogs at oral doses through 1500 mg/kg for 28 days, PTC124 has shown good tolerability. [1] The minimal concentration of PTC124 showing discernable readthrough was 0.01–0.1 mM (2.8–28 ng ml-1), whereas the concentration promoting maximal activity was approximately 3 mM(852 ng ml-1). [1] 3​ References: [1] Welch EM et al. Nature. 2007 May 3;447(7140):87-91 [2] Ming Du et al.PNAS. 2008 February 12;105(6):2064-2069 [3] Samit Hirawat et al. J Clin Pharmacol. 2007 Apr;47(4):430-44 The pharmacological and toxicological properties of this product have not been fully investigated. Exercise caution in use and handling. This product must not be used in humans.