The National Academy of Sciences (PNAS) have validated Translarna’s benefit

Today,  PTC Therapeutics announced that the Proceedings of the National Academy of Sciences (PNAS) have published further results to validate Translarna’s (ataluren) mechanism of action. Translarna proposes to promote read-through of premature stop codons which have resulted  from nonsense mutations in genetic disorders. The results reported in PNAS validate Ataluren’s ability to alter protein production. It is proposed that Ataluren inserts specific amino acids at premature codons, producing a full-length functional protein. “These new results help us better understand ataluren’s mechanism of action as well as confirm its protein restoration effect in genetic disorders,” said Stuart W. Peltz, Ph.D., co-founder and Chief Executive Officer of PTC Therapeutics. “These results further support our clinical findings demonstrating the production of full-length functional protein in nonsense mutation Duchenne muscular dystrophy and cystic fibrosis. Given this mechanism, ataluren offers the potential for a new therapeutic approach for multiple nonsense mutation genetic disorders by targeting the underlying cause of the disease.”