PTC Therapeutics, United Nations Educational, Scientific, and Cultural Organization | GenomeWeb

NJ Biotech’s Compound May Bypass Stop Mutations, Trigger Protein Expression

A small biotech company in New Jersey may soon begin human trials for a drug candidate that works by counteracting premature stop mutations believed to be linked to cystic fibrosis and muscular dystrophy.

The experimental compound, PTC124, was designed to manufacture a version of a protein that would otherwise not be expressed due to the mutation.

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