PTC124

PTC124 is a novel small-molecular agent that makes ribosomes less sensitive to stop codons. However, it is not equally effective with every stop codon, working best on the sequence 'UGA'.

It has been investigated for use against conditions caused by nonsense mutations, such as Duchenne muscular dystrophy and cystic fibrosis.

PTC124 is scheduled to enter the final phase of clinical trials in 2007.

It has been developed by PTC Therapeutics.