PTC-124(Ataluren)|775304-57-9-ShangHai Biochempartner Co.,Ltd.

PTC124 targets genetic disorders caused by nonsense mutations. PTC124 is an orally bioavailable compound that promotes of the human CFTR-G542X nonsense allele in a CF mouse model. Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.
PTC124 induces dose-dependent suppression of premature translational termination without concomitant effects on normal termination or mRNA decay.
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.
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).

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3.^ http://ptct.client.shareholder.com/releasedetail.cfm?ReleaseID=448803
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