Scientists in our Research and Early Development (RED) group held a poster session for employees, highlighting early-stage research with the potential to help lead to breakthrough scientific findings. Thanks to all of our researchers for showcasing the cutting-edge science helping us translate genetic discoveries into transformative medicines.
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We’re dedicated to understanding the unique experiences of children with achondroplasia. Our Lifetime Impact Study for Achondroplasia (LISA) identified real-world challenges these children face every day.
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This #RareDiseaseDay, we’re pleased to announce our annual RARE Scholars program for students with achondroplasia, Batten disease, hemophilia A, MPS or PKU is now accepting applications for the 2024-25 academic year.
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When Jess Klass discovered her newborn son, Matthias, was diagnosed with hemophilia A, she felt empowered with the knowledge she had from growing up with a brother who also lives with this inherited bleeding disorder. Read more about how Jess's personal experience fuels her work.
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