Nonsense mutations cause about 10-12% of cases across over 10,000 rare diseases, impacting roughly 40 million people. These mutations often lead to the most severe disease outcomes. 

At Nonsense Tx, we focus on developing small-molecule therapies to boost collagen 7 production for Dystrophic EB patients. 35% of RDEB patients have at least one nonsense mutation.

Early lab results using primary patient cells—tested with a leading expert on nonsense mutations in EB—have shown very promising outcomes

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