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United States : New Research validates Translarna's (ataluren) Mechanism of Action to Promote Readthrough of Nonsense Mutations and Produce Full-length Functional Protein.

PTC Therapeutics, Inc. today announced that the Proceedings of the National Academy of Sciences (PNAS) has published new results further validating Translarna's (ataluren) mechanism of action to promote readthrough of premature stop codons resulting from nonsense mutations in genetic disorders. The results reported in PNAS establish ataluren's ability to alter the protein production process at premature stop codons and to promote the insertion of specific amino acids and restore the production of 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."

The results published by PTC Therapeutics, Dr. Allan Jacobson and his team at the University of Massachusetts Medical School, and Dr. David Bedwell and his team at the University of Alabama, demonstrate ataluren treatment produces a protein that is similar to the protein from cells that do not have a nonsense mutation. The findings were verified in multiple nonsense mutation models. In addition, there have been almost 40 publications to date, many by independent investigators, demonstrating the clinical activity of Translarna across a spectrum of rare diseases.

"These data provide new insight on ataluren's effect on protein production and validates that it targets the source of nonsense mutation genetic disorders," said Allan Jacobson, Ph.D., co-founder and Board member of PTC Therapeutics and the Gerald L. Haidak, MD and Zelda S. Haidak professor of cell biology and chair of microbiology and physiological systems at University of Massachusetts Medical School. "Therapeutic nonsense suppression is a potentially powerful approach for the treatment of the large number of genetic disorders caused by nonsense mutations."

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Publication:Mena Report
Date:Oct 5, 2016
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