PTC124 Featured at Third Annual Congress ...(the symposium also announced that the Ph...)

Genetic disorders, such as DMD and cystic fibrosis (CF), are caused by genetic alterations, known as mutations. By targeting a specific type of genetic alteration -- nonsense mutations -- PTC124 bypasses the defect and leads to the restoration of a functional protein. The company has catalogued over 2,400 distinct genetic disorders for which nonsense mutations are the cause of the disease in a significant percentage of patients. Nonsense mutations inactivate gene function and are known to cause anywhere from five to 70 percent of the individual cases of most of these inherited diseases.

In preclinical models of genetic diseases harboring nonsense mutations, PTC124 was shown to regulate post-transcriptional control processes by restoring full-length, functional proteins. Post-transcriptional control processes are the cellular regulatory events that take place after an RNA molecule is copied from DNA. These processes are critical to proper cellular function and provide an opportunity for therapeutic intervention in a broad range of genetic disorders through the modulation of protein levels.

"We are pleased to have the opportunity to share our exciting clinical progress regarding PTC124 with the myology community," said Langdon Miller, M.D., Chief Medical Officer of PTC. "Through its specificity and novel mechanism of action, PTC124 has shown potential to address a broad range of genetic disorders due to nonsense mutations. We look forward to continuing to advance our clinical programs in DMD and CF and evaluating PTC124 in a number of additional nonsense-mutation-mediated genetic disorders."

About DMD/BMD

Duchenne and Becker muscular dystrophy (DMD/BMD) are progressive muscle di
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