Our team rang the opening bell at Nasdaq prior to our Investor Day event, where we updated Wall Street on our strategic vision for the future.
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We're pleased to join our partners CAMP4 Therapeutics in announcing a new research collaboration aimed at advancing novel therapeutics that increase protein levels by targeting regulatory RNA (regRNA) sequences, which are key elements controlling gene expression.
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Meet F8: a gene that plays a key role in blood clotting by telling cells how to make a protein called factor VIII. Changes in a person’s F8 gene can cause hemophilia A, a genetic condition that can lead to health complications, including uncontrolled bleeding and irreversible joint damage. Our scientists have led research into F8 with the goal of targeting the underlying cause of hemophilia A.
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No matter how someone grows, we all "rise with love." That's the underlying message behind Rising Love - a children's book that uses that analogy of baking bread to describe the experience of growing up with achondroplasia. Just as bread dough might rise to different shapes, sizes, and textures, bones grow uniquely in children with achondroplasia, the most common form of dwarfism.
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